LymphangiomaNCT06943703
Bleomycin as an Effective and Curative Adjunct Therapy for Lymphangioma
An observational study of Lymphangioma and Lymphatic Malformations, run by National Children's Hospital, Vietnam at 1 site in Vietnam.
Trial summary
Lymphatic malformations (LM), or lymphangiomas, are common vascular anomalies in children, most often occurring in the head and neck, less commonly in the axilla, abdomen, thorax, groin, and extremities. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Lymphangioma, Lymphatic Malformations
- What is being tested?
- Sclerotherapy with intralesional bleomycin injection
- Type of treatment
- Injection or infusion
- How many people
- 809 participants
- Where
- 1 siteVietnam
- Study dates
- 1 Jan 2018to 4 Feb 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The population of pediatric patients for this study will consist of children under the age of 18 who have been diagnosed with lymphangiomas (lymphatic malformations). These patients will have a variety of lesion locations, including the head and neck, axilla (armpit), abdomen, thorax (chest), groin, or extremities. The age range will encompass infants, children, and adolescents. They might or might not have received previous treatment with Bleomycin or other interventions for lymphatic malformations. Additionally, they might or might not serious comorbidities such as uncontrolled systemic diseases or immunosuppressive conditions. The cohort will be selected from the patient population at the National Children's Hospital, and participants will be enrolled based on a confirmed diagnosis of lymphangioma from 2018 to 2022.
Who may be able to join
- Age: Pediatric patients under the age of 18 years.
- Diagnosis: Confirmed diagnosis of lymphangioma (lymphatic malformation).
- Location of Lesions: Lymphangiomas located in the head and neck, axilla, abdomen, thorax, groin, or extremities.
- Treatment History: No prior treatment with Bleomycin for lymphangioma.
- Health Status: No serious comorbidities or medical conditions that could interfere with the study or treatment process.
- Informed Consent: Patients (or their legal guardians) provide informed consent to participate in the study.
Who cannot join
- Age: Patients over the age of 18 years.
- Pregnancy: Pregnant or breastfeeding patients.
- Other Treatments: Patients who have received prior treatments with Bleomycin for lymphangioma.
- Severe Comorbidities: Presence of serious comorbidities (e.g., uncontrolled cardiovascular disease, active infections) that may interfere with the study or treatment process.
- Allergy: Known allergy or hypersensitivity to Bleomycin or any of its components.
- Active Infections: Patients with active local or systemic infections at the time of enrollment.
- Immunosuppression: Immunocompromised patients (due to illness or medications).
Trial locations and contacts
1 site in 1 country.
Vietnam1 site
Department of Surgery, The National Children Hospital, Hanoi
Hanoi
About this study
Official title: Safety, Efficacy, and Long-Term Outcomes of Intralesional Bleomycin Injection as an Adjunctive Therapy for Pediatric Lymphatic Malformations: A Retrospective Study on Cosmetic and Health-Related Quality of Life Improvements.
Lymphatic malformations (LM), or lymphangiomas, are common vascular anomalies in children, most often occurring in the head and neck, less commonly in the axilla, abdomen, thorax, groin, and extremities. These benign but locally invasive lesions can cause complications such as respiratory issues, difficulty swallowing, nerve compression, bleeding, and cosmetic disfigurement. Treatment traditionally involves surgical excision, but this approach carries risks such as nerve damage, high recurrence, and poor cosmetic results. In recent years, sclerotherapy, specifically Bleomycin injection, has emerged as a less invasive alternative, offering potential benefits like reduced recurrence and better cosmetic outcomes.
Since its introduction in Vietnam in 2016, Bleomycin has been used to treat LM, but comprehensive studies on its safety, efficacy, and long-term outcomes in pediatric patients are lacking. This study aims to fill this gap by evaluating the effectiveness of intralesional Bleomycin injections in children with LM treated at the National Children's Hospital between 2018 and 2022.
Read more
The study will assess the safety, efficacy, and long-term outcomes of Bleomycin, focusing on lesion size reduction, recurrence rates, and cosmetic and health-related quality of life (QoL) improvements. The retrospective study will involve more than 800 pediatric patients and use tools like the Patient Scar Assessment Questionnaire (PSAQ) and Short Form-36 (SF-36) health survey to evaluate cosmetic outcomes and overall well-being.
The research will also compare the effectiveness of Bleomycin alone versus in combination with surgery, examining whether Bleomycin can reduce the need for additional interventions. By offering a comprehensive evaluation of both clinical and patient-reported outcomes, the study aims to establish Bleomycin as a viable, minimally invasive treatment option for pediatric LM, improving both clinical and cosmetic outcomes for these patients.
In addition, this study will provide valuable local data on Bleomycin's use in Vietnam, potentially influencing national treatment guidelines. The results will be disseminated in peer-reviewed journals, contributing to global understanding of Bleomycin's role in managing lymphatic malformations in children.
Read the detailed description
Lymphatic malformations (LM), often referred to as lymphangiomas, are a form of vascular malformation that arises due to the abnormal development of the lymphatic system. These benign but locally invasive lesions are one of the most common vascular anomalies in children. The highest incidence of lymphangiomas is seen in the head and neck region, where they can cause a range of serious complications such as respiratory obstruction, dysphagia (difficulty swallowing), and nerve compression. Less commonly locations of lymphangiomas include axilla, abdomen, thorax, groin, and extremities which could cause bleeding, compression of adjacent structures, and visible deformity, pain and discomfort. Left untreated, these malformations can lead to significant morbidity and functional impairment, as well as severe cosmetic disfigurement, particularly in pediatric patients.
Historically, surgical excision has been the primary treatment for lymphangiomas. However, this approach carries several risks, including damage to surrounding vital structures such as nerves and blood vessels, high recurrence rates due to incomplete excision, and unsightly scars. Over the last few decades, sclerotherapy (ST) has emerged as a less invasive, more favorable alternative that reduces morbidity while achieving superior cosmetic outcomes. Among various sclerosing agents, Bleomycin has gained popularity due to its mechanism of disrupting cellular functions in the abnormal lymphatic endothelial cells, leading to regression of the lesion.
Since its introduction to Vietnam in 2016, Bleomycin has been increasingly used as an adjunctive therapy for lymphangiomas. Despite its growing clinical application, there is a significant lack of comprehensive studies evaluating the safety, efficacy, and long-term outcomes of Bleomycin sclerotherapy in treating pediatric lymphatic malformations, particularly in the Vietnamese population. To date, no large-scale studies have assessed the clinical impact and quality-of-life outcomes of Bleomycin treatment for LM in children within the Vietnamese context. This study seeks to fill this gap in knowledge by comprehensively evaluating the long-term outcomes of intralesional Bleomycin injections in pediatric patients with lymphatic malformations treated at the National Children's Hospital in Vietnam.
The primary objective of this study is to evaluate the safety, efficacy, and long-term outcomes of intralesional Bleomycin injection for pediatric patients diagnosed with lymphatic malformations. The research will focus on both clinical success (such as reduction in lesion size and recurrence rates) and the cosmetic and health-related quality of life (QoL) improvements observed in the patients post-treatment. The study will also examine whether Bleomycin can effectively reduce the need for additional surgical interventions, thus improving both functional and aesthetic outcomes for these children.
This retrospective study will involve a cohort of more than 800 pediatric patients diagnosed with lymphatic malformations and treated with Bleomycin injections at the National Children's Hospital between 2018 and 2022. These patients will be evaluated for safety and efficacy by reviewing their medical records, monitoring for complications, and assessing the recurrence rates of lymphangiomas after treatment. Additionally, long-term cosmetic outcomes will be evaluated using validated patient-reported measures, such as the Patient Scar Assessment Questionnaire (PSAQ), and health-related quality of life will be assessed using the Short Form-36 (SF-36) health survey.
The study aims to assess the safety and efficacy of Bleomycin injection in pediatric patients diagnosed with lymphatic malformations at the National Children's Hospital between 2018 and 2022. This will be done by focusing on post-treatment complications and recurrence rates. Furthermore, the research intends to evaluate the long-term clinical outcomes, including lesion regression, recurrence, and the need for additional interventions. This will help assess the impact of Bleomycin on lesion size, post-treatment complication rates, and the overall clinical success of the intervention.
Another important goal of the study is to analyze the cosmetic outcomes and health-related quality of life in pediatric patients treated with Bleomycin sclerotherapy. The study will use standardized assessment tools like the PSAQ for scarring and the SF-36 for overall health outcomes. These tools will help quantify the patient-reported perception of treatment effects, including both cosmetic outcomes (scar appearance and symptoms) and overall well-being.
Additionally, the study will investigate the role of Bleomycin as a standalone treatment or in combination with surgical interventions to enhance treatment success while minimizing risks and complications associated with invasive procedures. By comparing these approaches, the research aims to determine if Bleomycin alone is sufficient or if a multimodal approach is more effective in treating lymphangiomas.
The results of this study will provide important insights into the feasibility and effectiveness of Bleomycin as an alternative to traditional surgical excision for the treatment of pediatric lymphangiomas. By examining both objective clinical outcomes and subjective patient-reported outcomes, such as quality of life and scar perception, this research will offer a comprehensive understanding of the benefits and challenges of using Bleomycin for this indication. The findings are expected to influence the clinical management of lymphangiomas in pediatric patients, offering evidence-based recommendations on the use of Bleomycin as a safe, effective, and cosmetically favorable treatment option.
Additionally, the study will contribute to filling the gap in local research on the use of Bleomycin for lymphatic malformations in Vietnam, a country where the clinical application of this agent has been relatively new and unstudied. The results of this research could lead to the establishment of evidence-based guidelines for Bleomycin treatment in the Vietnamese healthcare system, supporting its wider adoption as a standard therapy for lymphangiomas.
The study will be conducted with a strict ethical framework, ensuring that all patients and their families provide informed consent before participation, and that their privacy and confidentiality are strictly maintained. Data will be anonymized and stored securely throughout the research process. The findings will be disseminated through peer-reviewed scientific journals, with a particular focus on journals related to pediatric surgery, dermatology, and vascular anomalies, which could help raise awareness of Bleomycin's potential as a viable therapeutic option in the management of lymphangiomas.
In conclusion, this study will not only provide critical clinical data on the efficacy and safety of Bleomycin for treating pediatric lymphangiomas, but it will also enhance our understanding of the long-term cosmetic outcomes and quality of life improvements associated with this treatment. By offering a minimally invasive, less risky alternative to surgical excision, Bleomycin could revolutionize the management of lymphatic malformations in children, improving their health outcomes and overall well-being while minimizing the physical and psychological impact of the disease.
- Conditions
- Lymphangioma, Lymphatic Malformations
- Being tested
- Sclerotherapy with intralesional bleomycin injection
- Keywords
- Bleomycin, Lymphangioma, Lymphatic malformations, Intralesional Bleomycin injection, Sclerotherapy, Quality of life, Pediatric vascular anomalies, Head and neck, Safety and efficacy, Cosmetic, Non-surgical, Abdominal, Axilla, Thorax
- Medical subject headings
- Lymphangioma, Lymphatic Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sclerotherapy with intralesional bleomycin injectionDrug
The intervention studied is intralesional Bleomycin injection, which involves the direct injection of Bleomycin, a chemotherapeutic agent, into the lymphangioma lesions. This minimally invasive procedure is aimed at reducing the size of the lymphangioma by disrupting the abnormal growth of lymphatic vessels. The dosage of Bleomycin is tailored based on the patient's body weight, typically ranging from 1 to 3 mg per kg of body weight per injection. The intervention is distinct from other treatments, such as surgical excision or other sclerotherapy agents, as it specifically utilizes Bleomycin's mechanism of action to induce tissue sclerosis and promote lesion regression without the need for invasive surgery. This intervention may require multiple treatment sessions, depending on the size, location of the lymphangioma, and individual's response to therapy, with the goal of achieving long-term effectiveness and improving cosmetic outcomes.
| Study group | Role | What they receive |
|---|---|---|
| Pediatric Patients with Lymphangiomas Treated with Intralesional Bleomycin | This cohort consists of pediatric patients under the age of 18, diagnosed with lymphangiomas (lymphatic malformations), treated at the National Children's Hospital between 2018 and 2022. The lymphangiomas in this group are most commonly located in the head and neck, but may also occur, though less frequently, in the axilla, abdomen, thorax, groin, and extremities. The patients included in this cohort were selected based on their diagnosis of lymphangiomas without serious comorbidities that could complicate treatment or outcomes of the sclerotherapy. These patients were managed or observed during the study period, and the cohort represents a diverse age range of children under 18. Receives: Drug: Sclerotherapy with intralesional bleomycin injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Lesion Size
The degree of lesion size change will be measured to assess the efficacy of intralesional Bleomycin injection. This will be evaluated through clinical examination and imaging techniques (e.g., ultrasound or MRI) to determine the extent of regression of the lymphangioma.
Measured: Through study completion, an average of 5 years
Recurrence Rate
This outcome will track the recurrence rate of lymphangiomas after treatment with Bleomycin. Recurrence is defined as the reappearance or regrowth of the lesion after an initial reduction in size following treatment.
Measured: Through study completion, an average of 5 years
Adverse Reactions
The safety of the Bleomycin treatment will be assessed by monitoring adverse reactions, including infection, bleeding, swelling, or allergic reactions at the injection site, as well as any systemic side effects.
Measured: 1 week after each injection
Number of Injections Required for Remission
The number of Bleomycin injections required to achieve remission (defined as significant size reduction or complete resolution of the lesion) will be recorded. This will help assess the treatment protocol's efficiency and the typical treatment burden for patients.
Measured: Through study completion, an average of 5 years
Proportion of patients requiring additional interventions
This outcome will track the proportion of patients requiring additional interventions (e.g., surgical excision or further sclerotherapy sessions) after the initial Bleomycin treatment. The goal is to determine if Bleomycin alone is sufficient to treat the lymphangiomas or if supplementary interventions are necessary.
Measured: Through study completion, an average of 5 years
PSAQ score - Cosmetic Outcomes and Scar Perception
Cosmetic outcomes and patient scar perception were evaluated using the Patient Scar Assessment Questionnaire (PSAQ). This validated tool includes multiple subscales assessing appearance, symptoms, consciousness, and satisfaction with appearance. Each subscale is scored on a 4-point Likert scale, with individual item scores ranging from 1 to 4, where lower scores indicate better cosmetic outcomes and greater patient satisfaction. The minimum total score is 18, and the maximum is 72, with lower total scores representing more favorable scar outcomes.
Measured: Through study completion, an average of 5 years
SF-36 score - Health-Related Quality of Life (QoL)
Health-related quality of life was assessed using the Short Form-36 Health Survey (SF-36), a validated instrument comprising 36 items across eight domains: physical functioning, role limitations due to physical health, role limitations due to emotional problems, energy/fatigue, emotional well-being, social functioning, pain, and general health perceptions. Scores for each domain range from 0 to 100, with higher scores indicating better health-related quality of life. Both individual domain scores and composite physical and mental component summary scores were analyzed.
Measured: Through study completion, an average of 5 years
Secondary outcomes (2)
Follow-Up Time
The duration of follow-up visits post-treatment will be tracked to monitor for recurrence or complications. Follow-up time will also help assess the long-term effectiveness of Bleomycin treatment.
Measured: Through study completion, an average of 5 years
Time to Regrowth of Lesion
This outcome will measure the time to regrowth of the lymphangioma lesion after initial Bleomycin treatment. The time period between treatment and the first signs of regrowth will be monitored to evaluate the durability of the treatment.
Measured: Through study completion, an average of 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2025
- Study start1 Jan 2018
- Primary completion31 Dec 2022
- Study completion4 Feb 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (13)Papers the sponsor has linked to this study.
- Other trials for LymphangiomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 809 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Children's Hospital, Vietnam
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nguyen Thanh Quang, National Children's Hospital, Vietnam
- Investigators
- Quang T Nguyen, M.D., Principal investigator, Department of Pediatric Surgery, The National Hospital of Pediatrics, Hanoi, Vietnam
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06943703
- Sponsor's protocol ID
- VUNI.2223.SG04
- Status last verified
- Apr 2025
- First submitted
- 6 Apr 2025
- Last updated
- 24 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Acevedo JL, Shah RK, Brietzke SE. Nonsurgical therapies for lymphangiomas: a systematic review. Otolaryngol Head Neck Surg. 2008 Apr;138(4):418-24. doi: 10.1016/j.otohns.2007.11.018.
PubMed 18359347Contopoulos-Ioannidis DG, Karvouni A, Kouri I, Ioannidis JP. Reporting and interpretation of SF-36 outcomes in randomised trials: systematic review. BMJ. 2009 Jan 12;338:a3006. doi: 10.1136/bmj.a3006.
PubMed 19139138Wassef M, Blei F, Adams D, Alomari A, Baselga E, Berenstein A, Burrows P, Frieden IJ, Garzon MC, Lopez-Gutierrez JC, Lord DJ, Mitchel S, Powell J, Prendiville J, Vikkula M; ISSVA Board and Scientific Committee. Vascular Anomalies Classification: Recommendations From the International Society for the Study of Vascular Anomalies. Pediatrics. 2015 Jul;136(1):e203-14. doi: 10.1542/peds.2014-3673. Epub 2015 Jun 8.
PubMed 26055853Porwal PK, Dubey KP, Morey A, Singh H, Pooja S, Bose A. Bleomycin Sclerotherapy in Lymphangiomas of Head and Neck: Prospective Study of 8 Cases. Indian J Otolaryngol Head Neck Surg. 2018 Mar;70(1):145-148. doi: 10.1007/s12070-017-1243-x. Epub 2018 Jan 8.
PubMed 29456959Ameh EA, Nmadu PT. Cervical cystic hygroma: pre-, intra-, and post-operative morbidity and mortality in Zaria, Nigeria. Pediatr Surg Int. 2001 Jul;17(5-6):342-3. doi: 10.1007/s003830000558.
PubMed 11527161Bawazir OA, Bawazir R, Bawazir A, Kausar N, Said H. Efficacy and Clinical Outcomes of Bleomycin in the Treatment of Lymphangiomas: A Multicenter Experience. Dermatol Surg. 2021 Jul 1;47(7):948-952. doi: 10.1097/DSS.0000000000002976.
PubMed 33625132Yang Y, Sun M, Ma Q, Cheng X, Ao J, Tian L, Wang L, Lei D. Bleomycin A5 sclerotherapy for cervicofacial lymphatic malformations. J Vasc Surg. 2011 Jan;53(1):150-5. doi: 10.1016/j.jvs.2010.07.019. Epub 2010 Sep 16.
PubMed 20843632Parashar G, Shankar G, Sahadev R, Santhanakrishnan R. Intralesional Sclerotherapy with Bleomycin in Lymphatic Malformation of Tongue an Institutional Experience and Outcomes. J Indian Assoc Pediatr Surg. 2020 Mar-Apr;25(2):80-84. doi: 10.4103/jiaps.JIAPS_2_19. Epub 2020 Jan 28.
PubMed 32139985Durani P, McGrouther DA, Ferguson MW. The Patient Scar Assessment Questionnaire: a reliable and valid patient-reported outcomes measure for linear scars. Plast Reconstr Surg. 2009 May;123(5):1481-1489. doi: 10.1097/PRS.0b013e3181a205de.
PubMed 19407619Ardicli B, Karnak I, Ciftci AO, Tanyel FC, Senocak ME. Sclerotherapy with bleomycin versus surgical excision for extracervical cystic lymphatic malformations in children. Surg Today. 2016 Jan;46(1):97-101. doi: 10.1007/s00595-015-1128-0. Epub 2015 Feb 15.
PubMed 25682445Upadhyaya VD, Bhatnagar A, Kumar B, Neyaz Z, Kishore JS, Sthapak E. Is multiple session of intralesional bleomycin mandatory for complete resolution of macrocystic lymphatic malformation? Indian J Plast Surg. 2018 Jan-Apr;51(1):60-65. doi: 10.4103/ijps.IJPS_154_17.
PubMed 29928081Baskin D, Tander B, Bankaoglu M. Local bleomycin injection in the treatment of lymphangioma. Eur J Pediatr Surg. 2005 Dec;15(6):383-6. doi: 10.1055/s-2005-872922.
PubMed 16418953Tiwari P, Pandey V, Bera RN, Sharma SP, Chauhan N. Bleomycin sclerotherapy in lymphangiomas of the head and neck region: a prospective study. Int J Oral Maxillofac Surg. 2021 May;50(5):619-626. doi: 10.1016/j.ijom.2020.09.008. Epub 2020 Oct 12.
PubMed 33059994
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Efficacy, and Long-Term Outcomes of Intralesional Bleomycin Injection as an Adjunctive Therapy for Pediatric Lymphatic Malformations: A Retrospective Study on Cosmetic and Health-Related Quality of Life Improvements.
- ClinicalTrials.gov ID
- NCT06943703
- Acronym / sponsor ID
- VUNI.2223.SG04
- Conditions
- Lymphangioma, Lymphatic Malformations
- Keywords
- Bleomycin, Lymphangioma, Lymphatic malformations, Intralesional Bleomycin injection, Sclerotherapy, Quality of life, Pediatric vascular anomalies, Head and neck
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 809 participants (actual)
- Lead sponsor
- National Children's Hospital, VietnamUniversity, hospital or other
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 4 Feb 2025 (actual)
- First posted
- 24 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Lymphatic malformations (LM), or lymphangiomas, are common vascular anomalies in children, most often occurring in the head and neck, less commonly in the axilla, abdomen, thorax, groin, and extremities. These benign but locally invasive lesions can cause complications such as respiratory issues, difficulty swallowing, nerve compression, bleeding, and cosmetic disfigurement. Treatment traditionally involves surgical excision, but this approach carries risks such as nerve damage, high recurrence, and poor cosmetic results. In recent years, sclerotherapy, specifically Bleomycin injection, has emerged as a less invasive alternative, offering potential benefits like reduced recurrence and better cosmetic outcomes.
Since its introduction in Vietnam in 2016, Bleomycin has been used to treat LM, but comprehensive studies on its safety, efficacy, and long-term outcomes in pediatric patients are lacking. This study aims to fill this gap by evaluating the effectiveness of intralesional Bleomycin injections in children with LM treated at the National Children's Hospital between 2018 and 2022.
The study will assess the safety, efficacy, and long-term outcomes of Bleomycin, focusing on lesion size reduction, recurrence rates, and cosmetic and health-related quality of life (QoL) improvements. The retrospective study will involve more than 800 pediatric patients and use tools like the Patient Scar Assessment Questionnaire (PSAQ) and Short Form-36 (SF-36) health survey to evaluate cosmetic outcomes and overall well-being.
The research will also compare the effectiveness of Bleomycin alone versus in combination with surgery, examining whether Bleomycin can reduce the need for additional interventions. By offering a comprehensive evaluation of both clinical and patient-reported outcomes, the study aims to establish Bleomycin as a viable, minimally invasive treatment option for pediatric LM, improving both clinical and cosmetic outcomes for these patients.
In addition, this study will provide valuable local data on Bleomycin's use in Vietnam, potentially influencing national treatment guidelines. The results will be disseminated in peer-reviewed journals, contributing to global understanding of Bleomycin's role in managing lymphatic malformations in children.
Detailed description
Lymphatic malformations (LM), often referred to as lymphangiomas, are a form of vascular malformation that arises due to the abnormal development of the lymphatic system. These benign but locally invasive lesions are one of the most common vascular anomalies in children. The highest incidence of lymphangiomas is seen in the head and neck region, where they can cause a range of serious complications such as respiratory obstruction, dysphagia (difficulty swallowing), and nerve compression. Less commonly locations of lymphangiomas include axilla, abdomen, thorax, groin, and extremities which could cause bleeding, compression of adjacent structures, and visible deformity, pain and discomfort. Left untreated, these malformations can lead to significant morbidity and functional impairment, as well as severe cosmetic disfigurement, particularly in pediatric patients.
Historically, surgical excision has been the primary treatment for lymphangiomas. However, this approach carries several risks, including damage to surrounding vital structures such as nerves and blood vessels, high recurrence rates due to incomplete excision, and unsightly scars. Over the last few decades, sclerotherapy (ST) has emerged as a less invasive, more favorable alternative that reduces morbidity while achieving superior cosmetic outcomes. Among various sclerosing agents, Bleomycin has gained popularity due to its mechanism of disrupting cellular functions in the abnormal lymphatic endothelial cells, leading to regression of the lesion.
Since its introduction to Vietnam in 2016, Bleomycin has been increasingly used as an adjunctive therapy for lymphangiomas. Despite its growing clinical application, there is a significant lack of comprehensive studies evaluating the safety, efficacy, and long-term outcomes of Bleomycin sclerotherapy in treating pediatric lymphatic malformations, particularly in the Vietnamese population. To date, no large-scale studies have assessed the clinical impact and quality-of-life outcomes of Bleomycin treatment for LM in children within the Vietnamese context. This study seeks to fill this gap in knowledge by comprehensively evaluating the long-term outcomes of intralesional Bleomycin injections in pediatric patients with lymphatic malformations treated at the National Children's Hospital in Vietnam.
The primary objective of this study is to evaluate the safety, efficacy, and long-term outcomes of intralesional Bleomycin injection for pediatric patients diagnosed with lymphatic malformations. The research will focus on both clinical success (such as reduction in lesion size and recurrence rates) and the cosmetic and health-related quality of life (QoL) improvements observed in the patients post-treatment. The study will also examine whether Bleomycin can effectively reduce the need for additional surgical interventions, thus improving both functional and aesthetic outcomes for these children.
This retrospective study will involve a cohort of more than 800 pediatric patients diagnosed with lymphatic malformations and treated with Bleomycin injections at the National Children's Hospital between 2018 and 2022. These patients will be evaluated for safety and efficacy by reviewing their medical records, monitoring for complications, and assessing the recurrence rates of lymphangiomas after treatment. Additionally, long-term cosmetic outcomes will be evaluated using validated patient-reported measures, such as the Patient Scar Assessment Questionnaire (PSAQ), and health-related quality of life will be assessed using the Short Form-36 (SF-36) health survey.
The study aims to assess the safety and efficacy of Bleomycin injection in pediatric patients diagnosed with lymphatic malformations at the National Children's Hospital between 2018 and 2022. This will be done by focusing on post-treatment complications and recurrence rates. Furthermore, the research intends to evaluate the long-term clinical outcomes, including lesion regression, recurrence, and the need for additional interventions. This will help assess the impact of Bleomycin on lesion size, post-treatment complication rates, and the overall clinical success of the intervention.
Another important goal of the study is to analyze the cosmetic outcomes and health-related quality of life in pediatric patients treated with Bleomycin sclerotherapy. The study will use standardized assessment tools like the PSAQ for scarring and the SF-36 for overall health outcomes. These tools will help quantify the patient-reported perception of treatment effects, including both cosmetic outcomes (scar appearance and symptoms) and overall well-being.
Additionally, the study will investigate the role of Bleomycin as a standalone treatment or in combination with surgical interventions to enhance treatment success while minimizing risks and complications associated with invasive procedures. By comparing these approaches, the research aims to determine if Bleomycin alone is sufficient or if a multimodal approach is more effective in treating lymphangiomas.
The results of this study will provide important insights into the feasibility and effectiveness of Bleomycin as an alternative to traditional surgical excision for the treatment of pediatric lymphangiomas. By examining both objective clinical outcomes and subjective patient-reported outcomes, such as quality of life and scar perception, this research will offer a comprehensive understanding of the benefits and challenges of using Bleomycin for this indication. The findings are expected to influence the clinical management of lymphangiomas in pediatric patients, offering evidence-based recommendations on the use of Bleomycin as a safe, effective, and cosmetically favorable treatment option.
Additionally, the study will contribute to filling the gap in local research on the use of Bleomycin for lymphatic malformations in Vietnam, a country where the clinical application of this agent has been relatively new and unstudied. The results of this research could lead to the establishment of evidence-based guidelines for Bleomycin treatment in the Vietnamese healthcare system, supporting its wider adoption as a standard therapy for lymphangiomas.
The study will be conducted with a strict ethical framework, ensuring that all patients and their families provide informed consent before participation, and that their privacy and confidentiality are strictly maintained. Data will be anonymized and stored securely throughout the research process. The findings will be disseminated through peer-reviewed scientific journals, with a particular focus on journals related to pediatric surgery, dermatology, and vascular anomalies, which could help raise awareness of Bleomycin's potential as a viable therapeutic option in the management of lymphangiomas.
In conclusion, this study will not only provide critical clinical data on the efficacy and safety of Bleomycin for treating pediatric lymphangiomas, but it will also enhance our understanding of the long-term cosmetic outcomes and quality of life improvements associated with this treatment. By offering a minimally invasive, less risky alternative to surgical excision, Bleomycin could revolutionize the management of lymphatic malformations in children, improving their health outcomes and overall well-being while minimizing the physical and psychological impact of the disease.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric Patients with Lymphangiomas Treated with Intralesional Bleomycin | – | Drug: Sclerotherapy with intralesional bleomycin injectionThis cohort consists of pediatric patients under the age of 18, diagnosed with lymphangiomas (lymphatic malformations), treated at the National Children's Hospital between 2018 and 2022. The lymphangiomas in this group are most commonly located in the head and neck, but may also occur, though less frequently, in the axilla, abdomen, thorax, groin, and extremities. The patients included in this cohort were selected based on their diagnosis of lymphangiomas without serious comorbidities that could complicate treatment or outcomes of the sclerotherapy. These patients were managed or observed during the study period, and the cohort represents a diverse age range of children under 18. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The population of pediatric patients for this study will consist of children under the age of 18 who have been diagnosed with lymphangiomas (lymphatic malformations). These patients will have a variety of lesion locations, including the head and neck, axilla (armpit), abdomen, thorax (chest), groin, or extremities. The age range will encompass infants, children, and adolescents. They might or might not have received previous treatment with Bleomycin or other interventions for lymphatic malformations. Additionally, they might or might not serious comorbidities such as uncontrolled systemic diseases or immunosuppressive conditions. The cohort will be selected from the patient population at the National Children's Hospital, and participants will be enrolled based on a confirmed diagnosis of lymphangioma from 2018 to 2022.
Key inclusion criteria (6 listed)
- Age: Pediatric patients under the age of 18 years.
- Diagnosis: Confirmed diagnosis of lymphangioma (lymphatic malformation).
- Location of Lesions: Lymphangiomas located in the head and neck, axilla, abdomen, thorax, groin, or extremities.
- Treatment History: No prior treatment with Bleomycin for lymphangioma.
- Health Status: No serious comorbidities or medical conditions that could interfere with the study or treatment process.
Key exclusion criteria (7 listed)
- Age: Patients over the age of 18 years.
- Pregnancy: Pregnant or breastfeeding patients.
- Other Treatments: Patients who have received prior treatments with Bleomycin for lymphangioma.
- Severe Comorbidities: Presence of serious comorbidities (e.g., uncontrolled cardiovascular disease, active infections) that may interfere with the study or treatment process.
- Allergy: Known allergy or hypersensitivity to Bleomycin or any of its components.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sclerotherapy with intralesional bleomycin injectionThe intervention studied is intralesional Bleomycin injection, which involves the direct injection of Bleomycin, a chemotherapeutic agent, into the lymphangioma lesions. This minimally invasive procedure is aimed at reducing the size of the lymphangioma by disrupting the abnormal growth of lymphatic vessels. The dosage of Bleomycin is tailored based on the patient's body weight, typically ranging from 1 to 3 mg per kg of body weight per injection. The intervention is distinct from other treatments, such as surgical excision or other sclerotherapy agents, as it specifically utilizes Bleomycin's mechanism of action to induce tissue sclerosis and promote lesion regression without the need for invasive surgery. This intervention may require multiple treatment sessions, depending on the size, location of the lymphangioma, and individual's response to therapy, with the goal of achieving long-term effectiveness and improving cosmetic outcomes. | Drug | Pediatric Patients with Lymphangiomas Treated with Intralesional Bleomycin |
Primary outcome
Change in Lesion Size
Time frame: Through study completion, an average of 5 years
The degree of lesion size change will be measured to assess the efficacy of intralesional Bleomycin injection. This will be evaluated through clinical examination and imaging techniques (e.g., ultrasound or MRI) to determine the extent of regression of the lymphangioma.
Recurrence Rate
Time frame: Through study completion, an average of 5 years
This outcome will track the recurrence rate of lymphangiomas after treatment with Bleomycin. Recurrence is defined as the reappearance or regrowth of the lesion after an initial reduction in size following treatment.
Adverse Reactions
Time frame: 1 week after each injection
The safety of the Bleomycin treatment will be assessed by monitoring adverse reactions, including infection, bleeding, swelling, or allergic reactions at the injection site, as well as any systemic side effects.
Number of Injections Required for Remission
Time frame: Through study completion, an average of 5 years
The number of Bleomycin injections required to achieve remission (defined as significant size reduction or complete resolution of the lesion) will be recorded. This will help assess the treatment protocol's efficiency and the typical treatment burden for patients.
Proportion of patients requiring additional interventions
Time frame: Through study completion, an average of 5 years
This outcome will track the proportion of patients requiring additional interventions (e.g., surgical excision or further sclerotherapy sessions) after the initial Bleomycin treatment. The goal is to determine if Bleomycin alone is sufficient to treat the lymphangiomas or if supplementary interventions are necessary.
PSAQ score - Cosmetic Outcomes and Scar Perception
Time frame: Through study completion, an average of 5 years
Cosmetic outcomes and patient scar perception were evaluated using the Patient Scar Assessment Questionnaire (PSAQ). This validated tool includes multiple subscales assessing appearance, symptoms, consciousness, and satisfaction with appearance. Each subscale is scored on a 4-point Likert scale, with individual item scores ranging from 1 to 4, where lower scores indicate better cosmetic outcomes and greater patient satisfaction. The minimum total score is 18, and the maximum is 72, with lower total scores representing more favorable scar outcomes.
SF-36 score - Health-Related Quality of Life (QoL)
Time frame: Through study completion, an average of 5 years
Health-related quality of life was assessed using the Short Form-36 Health Survey (SF-36), a validated instrument comprising 36 items across eight domains: physical functioning, role limitations due to physical health, role limitations due to emotional problems, energy/fatigue, emotional well-being, social functioning, pain, and general health perceptions. Scores for each domain range from 0 to 100, with higher scores indicating better health-related quality of life. Both individual domain scores and composite physical and mental component summary scores were analyzed.
2 secondary outcomes
Follow-Up Time
Time frame: Through study completion, an average of 5 years
Time to Regrowth of Lesion
Time frame: Through study completion, an average of 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2025.
- Background: PubMed 18359347Acevedo JL, Shah RK, Brietzke SE. Nonsurgical therapies for lymphangiomas: a systematic review. Otolaryngol Head Neck Surg. 2008 Apr;138(4):418-24. doi: 10.1016/j.otohns.2007.11.018.
- Background: PubMed 19139138Contopoulos-Ioannidis DG, Karvouni A, Kouri I, Ioannidis JP. Reporting and interpretation of SF-36 outcomes in randomised trials: systematic review. BMJ. 2009 Jan 12;338:a3006. doi: 10.1136/bmj.a3006.
- Background: PubMed 26055853Wassef M, Blei F, Adams D, Alomari A, Baselga E, Berenstein A, Burrows P, Frieden IJ, Garzon MC, Lopez-Gutierrez JC, Lord DJ, Mitchel S, Powell J, Prendiville J, Vikkula M; ISSVA Board and Scientific Committee. Vascular Anomalies Classification: Recommendations From the International Society for the Study of Vascular Anomalies. Pediatrics. 2015 Jul;136(1):e203-14. doi: 10.1542/peds.2014-3673. Epub 2015 Jun 8.
- Results publication: PubMed 29456959Porwal PK, Dubey KP, Morey A, Singh H, Pooja S, Bose A. Bleomycin Sclerotherapy in Lymphangiomas of Head and Neck: Prospective Study of 8 Cases. Indian J Otolaryngol Head Neck Surg. 2018 Mar;70(1):145-148. doi: 10.1007/s12070-017-1243-x. Epub 2018 Jan 8.
- Results publication: PubMed 11527161Ameh EA, Nmadu PT. Cervical cystic hygroma: pre-, intra-, and post-operative morbidity and mortality in Zaria, Nigeria. Pediatr Surg Int. 2001 Jul;17(5-6):342-3. doi: 10.1007/s003830000558.
- Results publication: PubMed 33625132Bawazir OA, Bawazir R, Bawazir A, Kausar N, Said H. Efficacy and Clinical Outcomes of Bleomycin in the Treatment of Lymphangiomas: A Multicenter Experience. Dermatol Surg. 2021 Jul 1;47(7):948-952. doi: 10.1097/DSS.0000000000002976.
- Results publication: PubMed 20843632Yang Y, Sun M, Ma Q, Cheng X, Ao J, Tian L, Wang L, Lei D. Bleomycin A5 sclerotherapy for cervicofacial lymphatic malformations. J Vasc Surg. 2011 Jan;53(1):150-5. doi: 10.1016/j.jvs.2010.07.019. Epub 2010 Sep 16.
- Results publication: PubMed 32139985Parashar G, Shankar G, Sahadev R, Santhanakrishnan R. Intralesional Sclerotherapy with Bleomycin in Lymphatic Malformation of Tongue an Institutional Experience and Outcomes. J Indian Assoc Pediatr Surg. 2020 Mar-Apr;25(2):80-84. doi: 10.4103/jiaps.JIAPS_2_19. Epub 2020 Jan 28.
- Results publication: PubMed 19407619Durani P, McGrouther DA, Ferguson MW. The Patient Scar Assessment Questionnaire: a reliable and valid patient-reported outcomes measure for linear scars. Plast Reconstr Surg. 2009 May;123(5):1481-1489. doi: 10.1097/PRS.0b013e3181a205de.
- Results publication: PubMed 25682445Ardicli B, Karnak I, Ciftci AO, Tanyel FC, Senocak ME. Sclerotherapy with bleomycin versus surgical excision for extracervical cystic lymphatic malformations in children. Surg Today. 2016 Jan;46(1):97-101. doi: 10.1007/s00595-015-1128-0. Epub 2015 Feb 15.
- Results publication: PubMed 29928081Upadhyaya VD, Bhatnagar A, Kumar B, Neyaz Z, Kishore JS, Sthapak E. Is multiple session of intralesional bleomycin mandatory for complete resolution of macrocystic lymphatic malformation? Indian J Plast Surg. 2018 Jan-Apr;51(1):60-65. doi: 10.4103/ijps.IJPS_154_17.
- Results publication: PubMed 16418953Baskin D, Tander B, Bankaoglu M. Local bleomycin injection in the treatment of lymphangioma. Eur J Pediatr Surg. 2005 Dec;15(6):383-6. doi: 10.1055/s-2005-872922.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Quang T Nguyen, M.D. principal investigator
Department of Pediatric Surgery, The National Hospital of Pediatrics, Hanoi, Vietnam
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lymphangioma and Lymphatic Malformations, run by National Children's Hospital, Vietnam at 1 site in Vietnam.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Vietnam. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Lesion Size (time frame: Through study completion, an average of 5 years). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06943703, which also lists 13 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2025.