A Study of Rusfertide in Adults With Polycythemia Vera (PV)

The PROMIS Fatigue SF 8a T-score will be obtained via the item-response theory (IRT) response-pattern scoring approach using the PROMIS Scoring Service available at https://www.assessmentcenter.net/ac_scoringservice. It is an National Institutes of Health (NIH)-developed fatigue survey of 8 items that asks the participant about fatigue experience and impact over the past 7 days. Each item will be scored on a 5-point Likert scale (1=never/not at all to 5=almost always/very much). Raw scores (range: 8-40) will be converted into standardized T scores (range: 34.1-80.9), with higher scores indicating greater fatigue severity. A negative change from baseline indicates an improvement.

The MFSAF version 4.0 (v4.0) TSS7 is a questionnaire developed by the Patient-Reported Outcome (PRO) Consortium's Myelofibrosis Working Group that focuses on the 7 core symptoms of myelofibrosis: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. It includes 7 items on a 0 (absent) to 10 (worst imaginable) numeric rating scale. A higher score indicates worse symptoms. A negative change from baseline indicates an improvement.

The PROMIS Fatigue SF 8a T-score will be obtained via the IRT response-pattern scoring approach using the PROMIS Scoring Service available at https://www.assessmentcenter.net/ac_scoringservice. It is a National Institutes of Health (NIH)-developed fatigue survey of 8 items that asks the participant about fatigue experience and impact over the past 7 days. Each item will be scored on a 5-point Likert scale (1=never/not at all to 5=almost always/very much). Raw scores (range: 8-40) will be converted into standardized T scores (range: 34.1-80.9), with higher scores indicating greater fatigue severity. Moderate or severe symptoms are defined as a baseline PROMIS Fatigue SF 8aT-score >/= 58.8. A negative change from baseline indicates an improvement.

The MFSAF v4.0 is a questionnaire developed by the PRO Consortium's Myelofibrosis Working Group that focused on the 7 core symptoms of myelofibrosis: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. Items are scored on a 0 (absent) to 10 (worst imaginable) numeric rating scale. The MFSAF TSS4 is the sum of four items in the MFSAF (fatigue, itching [pruritis], night sweats, and abdominal discomfort). The weekly TSS4 will be calculated by averaging daily scores over a 7-day interval leading to Week 32 (inclusive). Moderate or severe symptoms are defined as a baseline MFSAF TSS4 >/=9.1. A higher score indicates worse symptoms. A negative change from baseline indicates an improvement.

MPN-SAF is a 10-item questionnaire measurement tool for symptoms of MPNs. Moderate or severe symptoms are defined as baseline MPN-SAF problems with concentration ≥4. The descriptive analysis approach the distribution of the MPN-SAF problems with concentration at Baseline will be categorized using the Patient Global Impression of Severity (PGI-S). The response quartiles will be used to establish the recommended cut-off points for the MPN-SAF Problems with Concentration corresponding to "none" or "mild" versus "moderate or "severe" categories on the PGI-S.

The PROMIS Fatigue SF 8a T-score will be obtained via the IRT response-pattern scoring approach using the PROMIS Scoring Service available at https://www.assessmentcenter.net/ac_scoringservice. It is an NIH-developed fatigue survey of 8 items that asks the participant about fatigue experience and impact over the past 7 days. Each item will be scored on a 5-point Likert scale (1=never/not at all to 5=almost always/very much). Raw scores (range: 8-40) will be converted into standardized T scores (range: 34.1-80.9), with higher scores indicating greater fatigue severity. Moderate or severe symptoms are defined as a baseline PROMIS Fatigue SF 8aT-score >/= 58.8.

The MFSAF v4.0 is a questionnaire developed by the PRO Consortium's Myelofibrosis Working Group that focuses on the 7 core symptoms of myelofibrosis: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. It included 7 items on a 0 (absent) to 10 (worst imaginable) numeric rating scale. The MFSAF TSS4 is the sum of four items in the MFSAF (fatigue, itching [pruritis], night sweats, and abdominal discomfort). The weekly TSS4 will be calculated by averaging daily scores over a 7-day interval leading to Week 32 (inclusive). Moderate or severe symptoms are defined as a baseline MFSAF TSS4 >/=9.1.

MPN-SAF is a 10-item questionnaire measurement tool for symptoms of MPNs. Moderate or severe symptoms are defined as baseline MPN-SAF problems with concentration ≥4. The descriptive analysis approach the distribution of the MPN-SAF problems with concentration at Baseline was categorized using the PGI-S. The response quartiles were used to establish the recommended cut-off points for the MPN-SAF Problems with Concentration corresponding to "none" or "mild" versus "moderate or "severe" categories on the PGI-S.

The PROMIS Fatigue SF 8a T-score will be obtained via the IRT response-pattern scoring approach using the PROMIS Scoring Service available at https://www.assessmentcenter.net/ac_scoringservice. It is an NIH-developed fatigue survey that includes 8 items that asks the participant about fatigue experience and impact over the past 7 days. Each item will be scored on a 5-point Likert scale (1=never/not at all to 5=almost always/very much). Raw scores (range: 8-40) will be converted into standardized T scores (range: 34.1-80.9), with higher scores indicating greater fatigue severity.

The PROMIS Fatigue SF 8a T-score was obtained via the IRT response-pattern scoring approach using the PROMIS Scoring Service available at https://www.assessmentcenter.net/ac_scoringservice. It is a National Institutes of Health (NIH)-developed fatigue survey of 8 items that asks the participant about fatigue experience and impact over the past 7 days. Each item will be scored on a 5-point Likert scale (1=never/not at all to 5=almost always/very much). Raw scores (range: 8-40) will be converted into standardized T scores (range: 34.1-80.9), with higher scores indicating greater fatigue severity. Moderate or severe symptoms are defined as a baseline PROMIS Fatigue SF 8aT-score >/= 58.8. A negative change from baseline indicates an improvement.

The MFSAF v4.0 is a questionnaire developed by the PRO Consortium's Myelofibrosis Working Group that focuses on the 7 core symptoms of myelofibrosis: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. It includes 7 items on a 0 (absent) to 10 (worst imaginable) numeric rating scale. A higher score indicates worse symptoms.

The MFSAF v4.0 is a questionnaire developed by the PRO Consortium's Myelofibrosis Working Group that focused on the 7 core symptoms of myelofibrosis: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. Items are scored on a 0 (absent) to 10 (worst imaginable) numeric rating scale. The MFSAF TSS4 is the sum of four items in the MFSAF (fatigue, itching [pruritis], night sweats, and abdominal discomfort). The weekly TSS4 will be calculated by averaging daily scores over a 7-day interval leading to Week 32 (inclusive). Moderate or severe symptoms are defined as a baseline MFSAF TSS4 >/=9.1. A higher score indicates worse symptoms. A negative change from baseline indicates an improvement.

The PROMIS Fatigue SF 8a T-score will be obtained via the IRT response-pattern scoring approach using the PROMIS Scoring Service available at https://www.assessmentcenter.net/ac_scoringservice. It is an NIH-developed fatigue survey that includes 8 items that asks the participant about fatigue experience and impact over the past 7 days. Each item will be scored on a 5-point Likert scale (1=never/not at all to 5=almost always/very much). Raw scores (range: 8-40) will be converted into standardized T scores (range: 34.1-80.9), with higher scores indicating greater fatigue severity. A negative change from baseline indicates an improvement.

The MFSAF v4.0 is a questionnaire developed by the PRO Consortium's Myelofibrosis Working Group that focuses on the 7 core symptoms of myelofibrosis: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. It includes 7 items on a 0 (absent) to 10 (worst imaginable) numeric rating scale. The MFSAF TSS4 is the sum of four items in the MFSAF (fatigue, itching [pruritis], night sweats, and abdominal discomfort). The weekly TSS4 will be calculated by averaging daily scores over a 7-day interval leading to Week 32 (inclusive).

MPN-SAF is a 10-item questionnaire measurement tool for symptoms of MPNs. Moderate or severe symptoms are defined as a baseline MPN-SAF problems with concentration ≥ 4. The descriptive analysis approach the distribution of the MPN-SAF problems with concentration at Baseline was categorized using the PGI-S. The response quartiles were used to establish the recommended cut-off points for the MPN-SAF Problems with Concentration corresponding to "none" or "mild" versus "moderate or "severe" categories on the PGI-S.

MPN-SAF is a 10-item questionnaire measurement tool for symptoms of MPNs. Moderate or severe symptoms are defined as a baseline MPN-SAF problems with concentration ≥ 4. The descriptive analysis approach the distribution of the MPN-SAF problems with concentration at Baseline was categorized using the PGI-S. The response quartiles were used to establish the recommended cut-off points for the MPN-SAF Problems with Concentration corresponding to "none" or "mild" versus "moderate or "severe" categories on the PGI-S.

The PGI-S is a single question to assess the severity of PV symptoms over the last 24 hours prior to dosing. The participant will determine the severity of their PV symptoms on a 4-point scale ranging from "None" (0) to "Severe" (3). A higher score indicates worse symptoms. A negative change from baseline indicates an improvement.

The PGI-S is a single question to assess the severity of PV symptoms over the last 24 hours prior to dosing. The participant will determine the severity of their PV symptoms on a 4-point scale ranging from "None" (0) to "Severe" (3). A higher score indicates worse symptoms. A negative change from baseline indicates an improvement.

PGI-C is a simple, commonly used instrument to obtain a participant's overall assessment of the impact of treatment on the participant's quality of life. Participants will compare his/her condition from the start of the study on a 5-point scale ranging from "Much better" (1) to "Much worse" (5) at the time of the assessment.

PGI-C is a simple, commonly used instrument to obtain a participant's overall assessment of the impact of treatment on the participant's quality of life. Participants will compare his/her condition from the start of the study on a 5-point scale ranging from "Much better" (1) to "Much worse" (5) at the time of the assessment. A higher score indicates a worse condition.

The MFSAF version 4.0 (v4.0) TSS7 is a questionnaire developed by the Patient-Reported Outcome (PRO) Consortium's Myelofibrosis Working Group that focuses on the 7 core symptoms of myelofibrosis: fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain. It includes 7 items on a 0 (absent) to 10 (worst imaginable) numeric rating scale. A higher score indicates worse symptoms. A negative change from baseline indicates an improvement.

The EORTC QLQ-C30 is a validated instrument to obtain a participant's overall assessment of the impact of treatment on the participant's quality of life. It consists of 5 functional scales (physical, role, cognitive, emotional, and social functioning), a global quality of life (QoL) scale, 3 symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (appetite loss, diarrhea, dyspnea, constipation, insomnia, financial impact). For the functional and the global QoL scales, a higher score indicates better health. For the symptom scales, a higher score indicates more symptom burden. The QLQ-C30 summary score is calculated as the mean of the combined 13 QLQ-C30 scale and item scores (excluding global QoL and financial impact), with a higher score indicating a better health related QoL. Raw scores are converted into scale scores ranging from 0 to 100.

Phlebotomy eligibility was defined as either: 1) A confirmed HCT ≥ 45% that was at least 3% higher than the baseline HCT, where confirmation was defined as 2 consecutive HCT assessments that were ≥ 45% and at least 3% higher than the baseline HCT, or 2) HCT ≥ 48%.

Phlebotomy eligibility was defined as either: 1) A confirmed HCT ≥ 45% that was at least 3% higher than the baseline HCT, where confirmation was defined as 2 consecutive HCT assessments that were ≥ 45% and at least 3% higher than the baseline HCT, or 2) HCT ≥ 48%.

Phlebotomy eligibility was defined as either: 1) A confirmed HCT ≥ 45% that was at least 3% higher than the baseline HCT, where confirmation was defined as 2 consecutive HCT assessments that were ≥ 45% and at least 3% higher than the baseline HCT, or 2) HCT ≥ 48%.

Phlebotomy eligibility was defined as either: 1) A confirmed HCT ≥ 45% that was at least 3% higher than the baseline HCT, where confirmation was defined as 2 consecutive HCT assessments that were ≥ 45% and at least 3% higher than the baseline HCT, or 2) HCT ≥ 48%.