Haplo-HSCT for Myelofibrosis
ConditionMyelofibrosis
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorPeking University People's Hospital
About this trial
Myelofibrosis (MF) is a myeloproliferative neoplasm causing bone marrow failure and high risk of leukemia transformation. JAK2 inhibitors improve symptoms but do not cure MF. Allogeneic stem cell transplantation (allo-HSCT) is the only potential cure, though limited donor availability restricts access. Haploidentical transplantation shows promise but associated with higher graft failure and treatment related mortality. We recently developed a novel regimen of haplo-SCT for MF. This study aims to investigate this novel protocol in a prospective trial to improve MF outcomes.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Primary disease type: Myelofibrosis (including primary myelofibrosis and myelofibrosis secondary to polycythemia vera or essential thrombocythemia).
No matched sibling donor or unrelated donor, with the availability of a haploidentical donor.
Signed informed consent.
Disqualifiers
Active infection
Very poor performance status (ECOG score > 2)
Estimated survival time < 30 days
Patient or family unable to cooperate
Trial design
Design model
Single group
Treatments tested in this trial
Haploidentical hematopoietic stem cell transplantation
Procedure/SurgeryThis is a single-arm study in which all patients will undergo haploidentical hematopoietic stem cell transplantation for the treatment of myelofibrosis. Pre-transplant Evaluation: Evaluation includes status of primary Disease, donor specific antibodies (DSA), organ function (assessments for heart, liver, lungs, kidneys, and the nervous system), and Spleen size. Transplantation Protocol: Conditioning Regimen: Dac/TT/Bu/Flu/ATG regimen: Decitabine 100 mg/m², on day -12. Thiotepa (TT) 5 mg/kg/day, on days -11 and -10. Busulfan 0.8 mg/kg body weight, every 6 hours, on days -8 to -6. Fludarabine 30 mg/m², once daily, on days -6 to -2. Anti-thymocyte globulin (ATG) 2.5 mg/kg body weight, once daily, on days -5 to -2. Transplant Donor: Haploidentical donor. GVHD (Graft-versus-Host Disease) Prophylaxis Regimen: Cyclosporine, mycophenolate mofetil, and short-course methotrexate for GVHD prevention. Graft: Target MNC (Mononuclear Cells): 6-8 × 10⁸/kg. Target CD34+ stem cells: 5 × 10⁶/kg
Treatment groups
39 Participants
are divided into 1 treatment groupGroup A: Haplo-SCT groupExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Cumulative incidence of Good graft function at day 60
The cumulative incidence of achieving both neutrophil engraftment and platelet engraftment at day 60 after Hematopoietic stem cell transplantation, independent of transfusion and granulocyte growth factor support, using competing risk model.
Time frame
Assessment at 60 days post-transplantationSecondary outcomes
1
Transplant-related mortality
TRM is defined as death due to any transplantation-related cause other than disease relapse. Transplant-related mortality will be calculated using a competing risks model.
Time frame
From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.2
overall survival
The time from hematopoietic stem cell transplantation to death from any cause in patients with myelofibrosis. Overall survival will be calculated using the Kaplan-Meier method.
Time frame
From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.3
Cumulative Incidence of Relapse
Relapse was defined as disease recurrence. The criteria for relapse include: 1. Increase in age-adjusted cellularity and abnormal M: E ratio. 2. Megakaryocytic abnormalities typical of MF (pleomorphism, hyperchromatic, cloud-like nuclei and megakaryocytic clusters). 3. Increase in grade of reticulin/collagen fibrosis (it should be borne in mind that the previously formed new bone usually takes a long time to be resorbed/resolved and that this should not therefore be used in grading post-transplant unless its density is significantly greater than the pre-transplant biopsy or there is active evidence of continuing new bone deposition).
Time frame
From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.4
Incidence of GVHD
The incidence refers to the rate at which new cases of GVHD occur within a timeframe. Acute GVHD was classified as symptom presentation before 100 days after alloHCT and chronic GVHD was classified as symptom presentation \>100 days after alloHCT. Each organ (skin, liver, and gut) was staged 1 through 4 for Acute (a) GVHD according to modified criteria based on the schema of the Mount Sinai Acute GVHD International Consortium (MAGIC), and patients were also assigned a grade of acute GVHD (I through IV) based on overall severity. Chronic (c) GVHD was graded in accordance with the National Institutes of Health (NIH) Chronic Graft-versus-Host Disease Consensus Criteria.
Time frame
From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov