Peking University People's Hospital
SUNY U before
Myelofibrosis (MF) is a myeloproliferative neoplasm causing bone marrow failure and a median survival of about 6 years. 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 is under-researched in MF. This study will validate its effectiveness in a prospective trial to improve MF outcomes.
Myelofibrosis
Haploidentical hematopoietic stem cell transplantation
Myelofibrosis (MF) is a myeloproliferative neoplasm characterized by clonal proliferation of hematopoietic stem cells, reactive hyperplasia of bone marrow stromal cells, and secondary inflammation and fibrosis, leading to progressive bone marrow failure and a high risk of acute myeloid leukemia transformation, with a median survival of about 6 years. While JAK2 inhibitors like ruxolitinib have been approved to improve symptoms and survival in MF patients, they do not provide a cure. Allogeneic stem cell transplantation (allo-HSCT) remains the only potential cure, but limited availability of matched sibling and unrelated donors often prevents patients from receiving this treatment. Haploidentical stem cell transplantation has shown good efficacy in leukemia but is less studied in MF, possibly due to concerns about graft failure, complications, and high transplant-related mortality. Our team has applied a mature haploidentical transplantation protocol for treating MF, which has shown promising results in preliminary observations. This study aims to further validate the effectiveness of this protocol through a prospective clinical trial, potentially establishing an effective approach for HSCT in MF and improving overall transplant outcomes.
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 39 participants |
| Official Title : | Haploidentical Hematopoietic Stem Cell Transplantation for the Treatment of Myelofibrosis: a Prospective, Single-center Study |
| Actual Study Start Date : | 2025-01-01 |
| Estimated Primary Completion Date : | 2027-12-31 |
| Estimated Study Completion Date : | 2027-12-31 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | |
| Sexes Eligible for Study: | ALL |
| Accepts Healthy Volunteers: |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
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