Rusfertide rapidly decreases hematocrit in patients with suboptimally controlled polycythemia vera.

Chew LP, Ginzburg YZ, Kirubamoorthy K, Lee SE, Lee JH, Modi NB, Khanna S, Dinh P, Valone F, Molina A, Gupta S

Leukemia research29 Oct 2025Cited by 2 (Europe PMC)

PMID41175501DOI10.1016/j.leukres.2025.108132PepBankPUB-41175501

Publisher page PubMedAsk about this study

Summary

Polycythemia vera (PV) is characterized by overproduction of erythrocytes, leukocytes, and platelets. Rusfertide is a hepcidin mimetic polypeptide that binds to ferroportin and decreases iron delivery to bone marrow, reducing the production of red blood cells. The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit. Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing. Of the 20 patients enrolled, 15 (75 %) completed 24 weeks of treatment, 13 (65 %) completed 52 weeks, and 7 discontinued rusfertide therapy. Mean baseline hematocrit was 51.6 %. By Week 8, 17 (85 %) patients achieved hematocrit < 45 %, and 19 (95 %) achieved ≥ 5 % absolute reduction from baseline. Median time to first hematocrit < 45 % was 4.9 weeks. Mean hematocrit was < 45 % at Week 4 and subsequently remained < 45 % throughout the study. No patients received phlebotomy during rusfertide treatment. Rusfertide led to a significant reduction in erythrocyte count (5.98 ×10¹²/L to 4.79 ×10¹²/L; nominal p-value 0.0036) and an improvement in iron deficiency markers (eg, ferritin and mean corpuscular volume) relative to baseline. Seventeen patients (85 %) experienced treatment-emergent adverse events, most of which were Grade 1/2. In this open-label phase 2 study, rusfertide resulted in a rapid and sustained improvement in hematocrit and was generally well tolerated. Rapid and sustained control of hematocrit reduces the need for therapeutic phlebotomy and may help reduce thrombotic and cardiovascular events over the long term in patients with PV.

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study9

9 results from studies that gave the peptide. 9 results from people.

01HumanNon-randomized trialOther
In patients with polycythemia vera, 7 discontinued rusfertide therapy.
In plain wordsIn patients with polycythemia vera, 7 stopped rusfertide treatment.
Peptide
Rusfertide
Outcome
Not applicableTreatment discontinuation
Study
n = 20
Outcome group
Adverse events · Whole body
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneousAs given in this study (human).
Measurements
Patients who discontinued rusfertide
7 patients
20 enrolled
Source sentences
Summary
Of the 20 patients enrolled, 15 (75 %) completed 24 weeks of treatment, 13 (65 %) completed 52 weeks, and 7 discontinued rusfertide therapy.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550101
02HumanNon-randomized trialEfficacy outcome
In patients with polycythemia vera, 17 (85 %) achieved hematocrit below 45 % by Week 8 with rusfertide.
In plain wordsIn patients with polycythemia vera, 85 % reached hematocrit below 45 % by Week 8 with rusfertide.
Peptide
Rusfertide
Outcome
Not applicableHematocrit below target
Study
n = 20
Outcome group
Blood counts and hematopoiesis · Whole body
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneous · 8 weeksAs given in this study (human).
Measurements
Patients with hematocrit <45% by Week 8
85%
rusfertide
Patients with hematocrit <45% by Week 8
17 patients
rusfertide
Source sentences
Summary
By Week 8, 17 (85 %) patients achieved hematocrit < 45 %, and 19 (95 %) achieved ≥ 5 % absolute reduction from baseline.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550102
03HumanNon-randomized trialEfficacy outcome
In patients with polycythemia vera, 19 (95 %) achieved at least 5 % absolute hematocrit reduction with rusfertide by Week 8.
In plain wordsIn patients with polycythemia vera, 95 % achieved at least 5 % absolute hematocrit reduction with rusfertide by Week 8.
Peptide
Rusfertide
Outcome
DecreaseHematocrit absolute reduction responder
Study
n = 20
Outcome group
Blood counts and hematopoiesis · Whole body
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneous · 8 weeksAs given in this study (human).
Measurements
Patients with >=5% absolute hematocrit reduction by Week 8
95%
rusfertide
Patients with >=5% absolute hematocrit reduction by Week 8
19 patients
rusfertide
Source sentences
Summary
By Week 8, 17 (85 %) patients achieved hematocrit < 45 %, and 19 (95 %) achieved ≥ 5 % absolute reduction from baseline.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550103
04HumanNon-randomized trialEfficacy outcome
No patients with polycythemia vera received phlebotomy during rusfertide treatment.
In plain wordsNo patients with polycythemia vera received blood removal treatment during rusfertide therapy.
Peptide
Rusfertide
Outcome
Not applicablePhlebotomy requirement
Study
n = 20
Outcome group
Blood counts and hematopoiesis · Cardiovascular
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneousAs given in this study (human).
Source sentences
Summary
No patients received phlebotomy during rusfertide treatment.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550106
05HumanNon-randomized trialSafety outcome
In patients with polycythemia vera, 85 % experienced treatment-emergent adverse events with rusfertide, mostly Grade 1/2.
In plain wordsIn patients with polycythemia vera, 85 % had adverse events during rusfertide treatment, mostly Grade 1/2.
Peptide
Rusfertide
Outcome
Not applicableTreatment-emergent adverse events
Study
n = 20
Outcome group
Adverse events · Whole body
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneousAs given in this study (human).
Measurements
Patients with treatment-emergent adverse events
85%
rusfertide-treated patients
Patients with treatment-emergent adverse events
17 patients
rusfertide-treated patients
Source sentences
Summary
Seventeen patients (85 %) experienced treatment-emergent adverse events, most of which were Grade 1/2.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550110
06HumanNon-randomized trialEfficacy outcome
In patients with polycythemia vera, median time to hematocrit below 45 % with rusfertide was 4.9 weeks.
In plain wordsIn patients with polycythemia vera, median time to hematocrit below 45 % with rusfertide was 4.9 weeks.
Peptide
Rusfertide
Outcome
Not applicableTime to hematocrit target
Study
n: not reported in source
Outcome group
Blood counts and hematopoiesis · Cardiovascular
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneousAs given in this study (human).
Measurements
Median time to first hematocrit <45%
4.9 weeks
patients with PV and baseline hematocrit >48%
Source sentences
Summary
Median time to first hematocrit < 45 % was 4.9 weeks.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550104
07HumanNon-randomized trialEfficacy outcome
In patients with polycythemia vera, mean hematocrit was below 45 % from Week 4 throughout rusfertide treatment.
In plain wordsIn patients with polycythemia vera, mean hematocrit stayed below 45 % from Week 4 during rusfertide treatment.
Peptide
Rusfertide
Outcome
Not applicableSustained mean hematocrit control
Study
n: not reported in source
Outcome group
Blood counts and hematopoiesis · Cardiovascular
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneousAs given in this study (human).
Measurements
Mean hematocrit
< 45%
from Week 4 through the study
Source sentences
Summary
Mean hematocrit was < 45 % at Week 4 and subsequently remained < 45 % throughout the study.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550105
08HumanNon-randomized trialEfficacy outcome
In patients with polycythemia vera, rusfertide significantly reduced erythrocyte count relative to baseline.
In plain wordsIn patients with polycythemia vera, rusfertide significantly reduced red blood cell count compared with baseline.
Peptide
Rusfertide
Outcome
DecreaseErythrocyte count
Study
n: not reported in source
Outcome group
Blood counts and hematopoiesis · Cardiovascular
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneousAs given in this study (human).
Measurements
Erythrocyte count
5.98 x10^12/L
baseline
Erythrocyte count
4.79 x10^12/L
after rusfertide treatment
Nominal p-value
0.0036
erythrocyte count change from baseline
Source sentences
Summary
Rusfertide led to a significant reduction in erythrocyte count (5.98 ×10¹²/L to 4.79 ×10¹²/L; nominal p-value 0.0036) and an improvement in iron deficiency markers (eg, ferritin and mean corpuscular volume) relative to baseline.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550107
09HumanNon-randomized trialEfficacy outcome
In patients with polycythemia vera, rusfertide was followed by improved iron deficiency markers (eg, ferritin and mean corpuscular volume) relative to baseline.
In plain wordsIn patients with polycythemia vera, iron deficiency markers such as ferritin and red blood cell size improved compared with baseline during rusfertide treatment.
Peptide
Rusfertide
Outcome
Direction not reportedIron deficiency markers (eg, ferritin and mean corpuscular volume)
Study
n: not reported in source
Outcome group
Blood biochemical markers · Cardiovascular
Studied dose
40 mg twice weekly until hematocrit target, then weekly dosing · subcutaneousAs given in this study (human).
Source sentences
Summary
Rusfertide led to a significant reduction in erythrocyte count (5.98 ×10¹²/L to 4.79 ×10¹²/L; nominal p-value 0.0036) and an improvement in iron deficiency markers (eg, ferritin and mean corpuscular volume) relative to baseline.
2 context sentences(design, population, supporting numbers)
Summary · context
The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit.
Summary · context
Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing.
Show in the study text Report a problemFND-4117550108

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Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
27 Oct 2026, 18:20 UTC
Study results updated
Version 1
27 Oct 2026, 16:32 UTC
First added · source update from Europe PMC

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