Haplo-HSCT for Myelofibrosis

NCT06674382 | Recruiting

• 2 other identifiers: 2024PHB400-001Peking University
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Myelofibrosis

Keywords

Haplo-HSCT; myelofibrosis; MF

Outcome Measures

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.

  Assessment at 60 days post-transplantation

Secondary Outcomes (11)

• Transplant-related mortality

  From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.

• overall survival

  From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.

• Cumulative Incidence of Relapse

  From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.

• Incidence of GVHD

  From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.

• Disease-free survival

  From baseline assessment at enrollment to the follow-up assessment at 36 months post-treatment.

Study Arms (1)

Haplo-SCT group

EXPERIMENTAL

Patients in this group will undergo haploidentical hematopoietic stem cell transplantation for the treatment of myelofibrosis.

Procedure: Haploidentical hematopoietic stem cell transplantation

Interventions

Haploidentical hematopoietic stem cell transplantation PROCEDURE

This 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

Haplo-SCT group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Active infection
• Very poor performance status (ECOG score \> 2)
• Estimated survival time \< 30 days
• Patient or family unable to cooperate
• Considered unsuitable after discussion

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University People's Hospital

Beijing, China

RECRUITING

Related Publications (3)

• Polverelli N, Hernandez-Boluda JC, Czerw T, Barbui T, D'Adda M, Deeg HJ, Ditschkowski M, Harrison C, Kroger NM, Mesa R, Passamonti F, Palandri F, Pemmaraju N, Popat U, Rondelli D, Vannucchi AM, Verstovsek S, Robin M, Colecchia A, Grazioli L, Damiani E, Russo D, Brady J, Patch D, Blamek S, Damaj GL, Hayden P, McLornan DP, Yakoub-Agha I. Splenomegaly in patients with primary or secondary myelofibrosis who are candidates for allogeneic hematopoietic cell transplantation: a Position Paper on behalf of the Chronic Malignancies Working Party of the EBMT. Lancet Haematol. 2023 Jan;10(1):e59-e70. doi: 10.1016/S2352-3026(22)00330-1. Epub 2022 Dec 6.

  PMID: 36493799 RESULT

• Cervantes F, Dupriez B, Passamonti F, Vannucchi AM, Morra E, Reilly JT, Demory JL, Rumi E, Guglielmelli P, Roncoroni E, Tefferi A, Pereira A. Improving survival trends in primary myelofibrosis: an international study. J Clin Oncol. 2012 Aug 20;30(24):2981-7. doi: 10.1200/JCO.2012.42.0240. Epub 2012 Jul 23.

  PMID: 22826273 RESULT

• Gangat N, Tefferi A. Myelofibrosis biology and contemporary management. Br J Haematol. 2020 Oct;191(2):152-170. doi: 10.1111/bjh.16576. Epub 2020 Mar 20.

  PMID: 32196650 RESULT

MeSH Terms

Conditions

Primary Myelofibrosis

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Sun Yuqian

CONTACT

861088326666; sunyuqian83@hotmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: November 3, 2024
• First Posted: November 5, 2024
• Study Start: January 1, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: May 14, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)