Hemophagocytic Lymphohistiocytosis (HLH)HSCT-HLHNCT07905066
Hemophagocytic Lymphohistiocytosis After Allogeneic Stem Cell Transplantation
An observational study of Hemophagocytic Lymphohistiocytosis (HLH), run by Peking University People's Hospital at 1 site in China.
Trial summary
Hemophagocytic lymphohistiocytosis (HLH) is a severe inflammatory complication that can occur after allogeneic hematopoietic stem cell transplantation. Its signs can overlap with those of infection and other transplant complications, making early recognition difficult. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophagocytic Lymphohistiocytosis (HLH)
- How many people
- 10,000 participants
- Where
- 1 siteChina
- Study dates
- 19 Oct 2026to 31 Dec 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults aged 18 years or older undergoing their first allogeneic hematopoietic stem cell transplantation at participating centers in China. The source population includes recipients who develop post-transplant HLH and those who do not. The retrospective component includes eligible transplantations from January 1, 2014, to the day before prospective study activation at each center. The prospective component consecutively enrolls eligible transplant recipients after site activation through December 31, 2029. A center- and transplant-year-stratified 15% random subcohort and all HLH cases are selected for detailed retrospective analysis.
Who may be able to join
- Age 18 years or older at first allogeneic hematopoietic stem cell transplantation.
- First allogeneic hematopoietic stem cell transplantation performed at a participating center within the study period.
- Verifiable transplantation date and basic clinical information. Written informed consent from the participant or legally authorized representative for prospective participation. Retrospective records are used under the applicable ethics-approved consent or waiver procedures.
Who cannot join
- Autologous transplantation or transplantation other than hematopoietic stem cell transplantation.
- HLH diagnosed before transplantation, or transplantation performed primarily for HLH.
- Previous allogeneic hematopoietic stem cell transplantation. Patient identity, transplantation date, or key outcome information cannot be verified.
- Explicit refusal to participate or to permit the use of clinical information for this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University People's Hospital
Beijing, Beijing Municipality, 100044
Xiaohui Zhang, MD, 01088326666, wujin1996@126.com
Xiao-Hui Zhang, MD
About this study
Official title: Hemophagocytic Lymphohistiocytosis After Hematopoietic Stem Cell Transplantation: A Multicenter Ambispective Longitudinal Cohort Study
Hemophagocytic lymphohistiocytosis (HLH) is a severe inflammatory complication that can occur after allogeneic hematopoietic stem cell transplantation. Its signs can overlap with those of infection and other transplant complications, making early recognition difficult. This study will examine whether changes in routine blood tests and clinical information can help identify patients who may develop HLH during the following seven days.
The study will combine a review of existing medical records with prospective follow-up at participating hospitals in China. Approximately 20,000 adults undergoing their first allogeneic transplantation will be included, with approximately 400 post-transplant HLH cases expected. Historical records will be used to develop the prediction model. Data from independent centers and prospectively enrolled patients will be used to validate the model.
Participants will be followed for one year after transplantation. Those who develop HLH during that year will also be followed for 90 days after HLH onset. The study will describe HLH occurrence, associated factors, treatments, and survival. All tests and treatments will remain part of routine care. No additional research samples or laboratory tests will be required, and model predictions will not be used to guide treatment.
Read the detailed description
This multicenter ambispective longitudinal observational cohort study will characterize de novo hemophagocytic lymphohistiocytosis (HLH) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) and develop and independently validate a dynamic model for HLH occurrence within the next seven days. Eligible participants are adults aged 18 years or older at their first allo-HSCT. Patients with HLH diagnosed before transplantation or transplantation performed primarily for HLH are excluded.
The retrospective component includes first allo-HSCT performed from January 1, 2014, through the day before prospective study activation at each center. Records available before activation will be reviewed. A stratified case-cohort design will be used: a 15% random subcohort will be selected within center and transplant-year strata, and all eligible HLH cases in the source cohort will be included in the detailed analysis. Patients belonging to both the random subcohort and the HLH case set will be counted once. Basic transplant and outcome information will be collected for the source cohort.
The prospective component will consecutively enroll eligible patients after ethics approval and site activation through December 31, 2029. Written informed consent will be obtained before transplantation, and participants will enter the transplant cohort after the procedure is completed. Additional research follow-up of retrospectively identified patients will require written informed consent. The planned combined source cohort comprises approximately 20,000 recipients, including approximately 400 expected HLH cases.
Data collection includes underlying disease and disease status, comorbidities, donor and transplant characteristics, conditioning, graft-versus-host disease prophylaxis, engraftment, infections, viral reactivation, post-transplant lymphoproliferative disease, organ dysfunction, and treatments. Longitudinal routine laboratory data include blood counts, ferritin, C-reactive protein, fibrinogen, triglycerides, D-dimer, lactate dehydrogenase, albumin, and liver and kidney function. Cytokines, soluble CD25, and natural killer cell function results will be recorded when obtained during routine care. Sampling times, result availability times, units, and reference ranges will be retained.
The primary clinical outcome is the first de novo HLH episode within 365 days after allo-HSCT. An episode must meet at least five of the eight HLH-2004 criteria during the same clinical episode and be confirmed by the adjudication group. Two investigators will independently review potential cases, with a third investigator resolving disagreement. The onset date is the earliest date on which the study definition is fulfilled. Dates of first clinical suspicion, clinical diagnosis, and HLH-directed treatment initiation will also be recorded. Clinically diagnosed episodes not meeting the primary definition will be recorded separately for supplementary analyses. Adjudicators will not have access to new-model predictions. Death and second allogeneic transplantation before HLH will be treated as competing events.
At evaluable assessment times from day 1 through day 358 after transplantation, information already available at that time will be used to predict first HLH onset within the subsequent seven days. Model evaluation will use assessments obtained before HLH onset and before initiation of HLH-directed treatment. Models and thresholds will be fixed before independent-center and prospective validation. Prospective predictions will be recorded for research and will not be provided to treating teams as treatment decision prompts.
Follow-up will continue through one year after transplantation. Clinical and outcome information will be reviewed monthly during the first six months and at months 9 and 12; routine inpatient data will be collected longitudinally. For participants who develop HLH during the first year, additional assessments will be recorded on days 7, 14, 28, 60, and 90 after HLH onset. Follow-up will use hospital and outpatient records, telephone, online or video contact, or in-person visits. Patients developing HLH late in the first year may be followed for up to approximately 15 months after transplantation.
Analyses will account for case-cohort sampling, competing events, center, and transplant period. Dynamic landmark models, penalized regression, and gradient boosting will be evaluated. Inflammatory characteristics will be examined using representative subcohort data and compared with clinical and simplified models. Performance will be assessed using discrimination, calibration, Brier scores, decision curves, case detection, positive predictive value, warning lead time, and false-alert frequency. Secondary analyses will describe HLH triggers, organ support, treatment patterns, recorded early treatment response, and survival.
The study does not assign treatment or require additional blood sampling, laboratory tests, or biospecimen retention. Historical records will be used under the applicable ethics-approved consent or waiver procedures; prospective participants will provide written informed consent. Each site will retain its identity linkage file. The coordinating database will receive coded data without direct personal identifiers, and results will be reported in aggregate.
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH)
- Keywords
- Allogeneic hematopoietic stem cell transplantation, Post-transplant HLH, Secondary hemophagocytic lymphohistiocytosis, Dynamic risk prediction, Longitudinal cohort, Case-cohort study
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Retrospective cohort | Patients whose first visit to our institution and the termination of follow-up both occurred before the opening of this study will contribute to the retrospective cohort. | |
| Prospective cohort | Patients whose first visit to our institution occur after the opening of this study will contribute to the prospective cohort. | |
| Retrospective/Prospective cohort | Patients whose first visit to our institution occurred before the opening of this study and whose follow-up will terminate after the opening of this study will contribute to the ambispective cohort. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative Incidence of First De Novo HLH Within 365 Days After Transplantation
Cumulative incidence of the first adjudicated de novo HLH episode after first allogeneic hematopoietic stem cell transplantation. HLH requires at least five of the eight HLH-2004 criteria within the same clinical episode and confirmation by the adjudication group. The onset date is the first date the study definition is met. Death and second allogeneic transplantation before HLH are competing events.
Measured: From transplantation through day 365.
Discrimination of the 7-Day HLH Risk Prediction Model
Time-dependent area under the receiver operating characteristic curve for predicting first HLH onset within the next seven days, evaluated separately in independent-center and prospective validation cohorts. Values range from 0 to 1, with higher values indicating better discrimination. Estimates account for the sampling design and follow-up.
Measured: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Calibration of Predicted 7-Day HLH Risk
Agreement between predicted seven-day probabilities and observed seven-day HLH occurrence, evaluated using calibration curves in independent-center and prospective validation cohorts. Observed occurrence estimates account for the sampling design, follow-up, and competing events.
Measured: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Positive Predictive Value of 7-Day HLH Alerts
Percentage of evaluable model-positive assessments followed by adjudicated first HLH onset within the next seven days. A positive assessment is defined using the prediction threshold fixed before validation. Estimates are adjusted for the sampling design and follow-up where applicable.
Measured: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Case Detection Rate for Incident HLH
Percentage of adjudicated incident HLH cases with at least one model-positive assessment during the seven days before onset, among cases with an eligible pre-onset model assessment. The prediction threshold is fixed before validation. Estimates are adjusted for the sampling design and follow-up where applicable.
Measured: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Secondary outcomes (5)
Cumulative Incidence of HLH Within 100 Days After Transplantation
Cumulative incidence of first adjudicated de novo HLH, using the same definition as the primary clinical outcome. Death and second allogeneic transplantation before HLH are competing events.
Measured: From transplantation through day 100.
All-Cause Mortality Within 28 Days After HLH Onset
Probability of death from any cause within 28 days after adjudicated HLH onset among participants who develop HLH. Follow-up time starts on the HLH onset date.
Measured: From HLH onset through day 28 after onset.
All-Cause Mortality Within 60 Days After HLH Onset
Probability of death from any cause within 60 days after adjudicated HLH onset among participants who develop HLH. Follow-up time starts on the HLH onset date.
Measured: From HLH onset through day 60 after onset.
All-Cause Mortality Within 90 Days After HLH Onset
Probability of death from any cause within 90 days after adjudicated HLH onset among participants who develop HLH. Follow-up time starts on the HLH onset date.
Measured: From HLH onset through day 90 after onset.
Overall Survival at One Year After Transplantation
Probability of survival one year after first allogeneic hematopoietic stem cell transplantation. Death from any cause is the event, and follow-up time starts on the transplantation date.
Measured: From transplantation through day 365.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2026
- Study start19 Oct 2026
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hemophagocytic Lymphohistiocytosis (HLH)Studies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- 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
- Other
- Enrolment
- 10,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
- Investigators
- Xiao-Hui Zhang, MD, Principal investigator, Peking University Institute of Hematology, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07905066
- Sponsor's protocol ID
- HSCT-HLH2026
- Acronym
- HSCT-HLH
- Status last verified
- Oct 2026
- First submitted
- 23 Oct 2026
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: There is no current plan to share individual participant data with external researchers. The study involves multicenter clinical records, and access to patient-level data is subject to applicable ethical approvals, institutional policies, and data-use agreements.
Publications
La Rosee P, Horne A, Hines M, von Bahr Greenwood T, Machowicz R, Berliner N, Birndt S, Gil-Herrera J, Girschikofsky M, Jordan MB, Kumar A, van Laar JAM, Lachmann G, Nichols KE, Ramanan AV, Wang Y, Wang Z, Janka G, Henter JI. Recommendations for the management of hemophagocytic lymphohistiocytosis in adults. Blood. 2019 Jun 6;133(23):2465-2477. doi: 10.1182/blood.2018894618. Epub 2019 Apr 16.
PubMed 30992265Henter JI, Horne A, Arico M, Egeler RM, Filipovich AH, Imashuku S, Ladisch S, McClain K, Webb D, Winiarski J, Janka G. HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2007 Feb;48(2):124-31. doi: 10.1002/pbc.21039.
PubMed 16937360
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hemophagocytic Lymphohistiocytosis After Hematopoietic Stem Cell Transplantation: A Multicenter Ambispective Longitudinal Cohort Study
- ClinicalTrials.gov ID
- NCT07905066
- Acronym / sponsor ID
- HSCT-HLH / HSCT-HLH2026
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH)
- Keywords
- Allogeneic hematopoietic stem cell transplantation, Post-transplant HLH, Secondary hemophagocytic lymphohistiocytosis, Dynamic risk prediction, Longitudinal cohort, Case-cohort study
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10,000 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 19 Oct 2026 (actual)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 29 Oct 2026 (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.
Hemophagocytic lymphohistiocytosis (HLH) is a severe inflammatory complication that can occur after allogeneic hematopoietic stem cell transplantation. Its signs can overlap with those of infection and other transplant complications, making early recognition difficult. This study will examine whether changes in routine blood tests and clinical information can help identify patients who may develop HLH during the following seven days.
The study will combine a review of existing medical records with prospective follow-up at participating hospitals in China. Approximately 20,000 adults undergoing their first allogeneic transplantation will be included, with approximately 400 post-transplant HLH cases expected. Historical records will be used to develop the prediction model. Data from independent centers and prospectively enrolled patients will be used to validate the model.
Participants will be followed for one year after transplantation. Those who develop HLH during that year will also be followed for 90 days after HLH onset. The study will describe HLH occurrence, associated factors, treatments, and survival. All tests and treatments will remain part of routine care. No additional research samples or laboratory tests will be required, and model predictions will not be used to guide treatment.
Detailed description
This multicenter ambispective longitudinal observational cohort study will characterize de novo hemophagocytic lymphohistiocytosis (HLH) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) and develop and independently validate a dynamic model for HLH occurrence within the next seven days. Eligible participants are adults aged 18 years or older at their first allo-HSCT. Patients with HLH diagnosed before transplantation or transplantation performed primarily for HLH are excluded.
The retrospective component includes first allo-HSCT performed from January 1, 2014, through the day before prospective study activation at each center. Records available before activation will be reviewed. A stratified case-cohort design will be used: a 15% random subcohort will be selected within center and transplant-year strata, and all eligible HLH cases in the source cohort will be included in the detailed analysis. Patients belonging to both the random subcohort and the HLH case set will be counted once. Basic transplant and outcome information will be collected for the source cohort.
The prospective component will consecutively enroll eligible patients after ethics approval and site activation through December 31, 2029. Written informed consent will be obtained before transplantation, and participants will enter the transplant cohort after the procedure is completed. Additional research follow-up of retrospectively identified patients will require written informed consent. The planned combined source cohort comprises approximately 20,000 recipients, including approximately 400 expected HLH cases.
Data collection includes underlying disease and disease status, comorbidities, donor and transplant characteristics, conditioning, graft-versus-host disease prophylaxis, engraftment, infections, viral reactivation, post-transplant lymphoproliferative disease, organ dysfunction, and treatments. Longitudinal routine laboratory data include blood counts, ferritin, C-reactive protein, fibrinogen, triglycerides, D-dimer, lactate dehydrogenase, albumin, and liver and kidney function. Cytokines, soluble CD25, and natural killer cell function results will be recorded when obtained during routine care. Sampling times, result availability times, units, and reference ranges will be retained.
The primary clinical outcome is the first de novo HLH episode within 365 days after allo-HSCT. An episode must meet at least five of the eight HLH-2004 criteria during the same clinical episode and be confirmed by the adjudication group. Two investigators will independently review potential cases, with a third investigator resolving disagreement. The onset date is the earliest date on which the study definition is fulfilled. Dates of first clinical suspicion, clinical diagnosis, and HLH-directed treatment initiation will also be recorded. Clinically diagnosed episodes not meeting the primary definition will be recorded separately for supplementary analyses. Adjudicators will not have access to new-model predictions. Death and second allogeneic transplantation before HLH will be treated as competing events.
At evaluable assessment times from day 1 through day 358 after transplantation, information already available at that time will be used to predict first HLH onset within the subsequent seven days. Model evaluation will use assessments obtained before HLH onset and before initiation of HLH-directed treatment. Models and thresholds will be fixed before independent-center and prospective validation. Prospective predictions will be recorded for research and will not be provided to treating teams as treatment decision prompts.
Follow-up will continue through one year after transplantation. Clinical and outcome information will be reviewed monthly during the first six months and at months 9 and 12; routine inpatient data will be collected longitudinally. For participants who develop HLH during the first year, additional assessments will be recorded on days 7, 14, 28, 60, and 90 after HLH onset. Follow-up will use hospital and outpatient records, telephone, online or video contact, or in-person visits. Patients developing HLH late in the first year may be followed for up to approximately 15 months after transplantation.
Analyses will account for case-cohort sampling, competing events, center, and transplant period. Dynamic landmark models, penalized regression, and gradient boosting will be evaluated. Inflammatory characteristics will be examined using representative subcohort data and compared with clinical and simplified models. Performance will be assessed using discrimination, calibration, Brier scores, decision curves, case detection, positive predictive value, warning lead time, and false-alert frequency. Secondary analyses will describe HLH triggers, organ support, treatment patterns, recorded early treatment response, and survival.
The study does not assign treatment or require additional blood sampling, laboratory tests, or biospecimen retention. Historical records will be used under the applicable ethics-approved consent or waiver procedures; prospective participants will provide written informed consent. Each site will retain its identity linkage file. The coordinating database will receive coded data without direct personal identifiers, and results will be reported in aggregate.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Retrospective cohort | – | –Patients whose first visit to our institution and the termination of follow-up both occurred before the opening of this study will contribute to the retrospective cohort. |
| Prospective cohort | – | –Patients whose first visit to our institution occur after the opening of this study will contribute to the prospective cohort. |
| Retrospective/Prospective cohort | – | –Patients whose first visit to our institution occurred before the opening of this study and whose follow-up will terminate after the opening of this study will contribute to the ambispective cohort. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults aged 18 years or older undergoing their first allogeneic hematopoietic stem cell transplantation at participating centers in China. The source population includes recipients who develop post-transplant HLH and those who do not. The retrospective component includes eligible transplantations from January 1, 2014, to the day before prospective study activation at each center. The prospective component consecutively enrolls eligible transplant recipients after site activation through December 31, 2029. A center- and transplant-year-stratified 15% random subcohort and all HLH cases are selected for detailed retrospective analysis.
Key inclusion criteria (3 listed)
- Age 18 years or older at first allogeneic hematopoietic stem cell transplantation.
- First allogeneic hematopoietic stem cell transplantation performed at a participating center within the study period.
- Verifiable transplantation date and basic clinical information. Written informed consent from the participant or legally authorized representative for prospective participation. Retrospective records are used under the applicable ethics-approved consent or waiver procedures.
Key exclusion criteria (4 listed)
- Autologous transplantation or transplantation other than hematopoietic stem cell transplantation.
- HLH diagnosed before transplantation, or transplantation performed primarily for HLH.
- Previous allogeneic hematopoietic stem cell transplantation. Patient identity, transplantation date, or key outcome information cannot be verified.
- Explicit refusal to participate or to permit the use of clinical information for this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Cumulative Incidence of First De Novo HLH Within 365 Days After Transplantation
Time frame: From transplantation through day 365.
Cumulative incidence of the first adjudicated de novo HLH episode after first allogeneic hematopoietic stem cell transplantation. HLH requires at least five of the eight HLH-2004 criteria within the same clinical episode and confirmation by the adjudication group. The onset date is the first date the study definition is met. Death and second allogeneic transplantation before HLH are competing events.
Discrimination of the 7-Day HLH Risk Prediction Model
Time frame: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Time-dependent area under the receiver operating characteristic curve for predicting first HLH onset within the next seven days, evaluated separately in independent-center and prospective validation cohorts. Values range from 0 to 1, with higher values indicating better discrimination. Estimates account for the sampling design and follow-up.
Calibration of Predicted 7-Day HLH Risk
Time frame: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Agreement between predicted seven-day probabilities and observed seven-day HLH occurrence, evaluated using calibration curves in independent-center and prospective validation cohorts. Observed occurrence estimates account for the sampling design, follow-up, and competing events.
Positive Predictive Value of 7-Day HLH Alerts
Time frame: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Percentage of evaluable model-positive assessments followed by adjudicated first HLH onset within the next seven days. A positive assessment is defined using the prediction threshold fixed before validation. Estimates are adjusted for the sampling design and follow-up where applicable.
Case Detection Rate for Incident HLH
Time frame: At evaluable assessments on days 1 to 358 after transplantation, with HLH occurrence evaluated over the subsequent 7 days, through day 365.
Percentage of adjudicated incident HLH cases with at least one model-positive assessment during the seven days before onset, among cases with an eligible pre-onset model assessment. The prediction threshold is fixed before validation. Estimates are adjusted for the sampling design and follow-up where applicable.
5 secondary outcomes
Cumulative Incidence of HLH Within 100 Days After Transplantation
Time frame: From transplantation through day 100.
All-Cause Mortality Within 28 Days After HLH Onset
Time frame: From HLH onset through day 28 after onset.
All-Cause Mortality Within 60 Days After HLH Onset
Time frame: From HLH onset through day 60 after onset.
All-Cause Mortality Within 90 Days After HLH Onset
Time frame: From HLH onset through day 90 after onset.
Overall Survival at One Year After Transplantation
Time frame: From transplantation through day 365.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Results publication: PubMed 30992265La Rosee P, Horne A, Hines M, von Bahr Greenwood T, Machowicz R, Berliner N, Birndt S, Gil-Herrera J, Girschikofsky M, Jordan MB, Kumar A, van Laar JAM, Lachmann G, Nichols KE, Ramanan AV, Wang Y, Wang Z, Janka G, Henter JI. Recommendations for the management of hemophagocytic lymphohistiocytosis in adults. Blood. 2019 Jun 6;133(23):2465-2477. doi: 10.1182/blood.2018894618. Epub 2019 Apr 16.
- Results publication: PubMed 16937360Henter JI, Horne A, Arico M, Egeler RM, Filipovich AH, Imashuku S, Ladisch S, McClain K, Webb D, Winiarski J, Janka G. HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2007 Feb;48(2):124-31. doi: 10.1002/pbc.21039.
- Individual participant dataSharing planned: No. There is no current plan to share individual participant data with external researchers. The study involves multicenter clinical records, and access to patient-level data is subject to applicable ethical approvals, institutional policies, and data-use agreements.
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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.
Xiao-Hui Zhang, MD principal investigator
Peking University Institute of Hematology, Peking University People's Hospital
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 Hemophagocytic Lymphohistiocytosis (HLH), run by Peking University People's Hospital at 1 site in China.
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 3 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 29 Oct 2026. 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 China. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jin Wu, MD (086-01088326002, wujin1996@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative Incidence of First De Novo HLH Within 365 Days After Transplantation (time frame: From transplantation through day 365.). The study lists 5 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 NCT07905066, which also lists 2 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 29 Oct 2026.