What does this research mean for the field?

Haploidentical haematopoietic stem cell transplantation is a highly effective curative option for patients with transfusion-dependent thalassaemia who lack fully matched donors, achieving over 90% 2-year overall and event-free survival. Novelty: ClaimNovelty.INCREMENTAL. Consensus alignment: ConsensusAlignment.NEUTRAL.

What question did this study set out to answer?

To assess the efficacy and safety of haploidentical haematopoietic stem cell transplantation in patients with transfusion-dependent thalassaemia lacking fully matched donors.

September 3, 2026British Journal of HaematologyOpen Access

Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase IV , open‐label, single‐centre trial

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Authors

XLXiaoxuan LaiSouthern University of Science and Technology RLRongrong LiuXuzhou Medical College HXHongwen XiaoFirst Affiliated Hospital of GuangXi Medical University

Discussion

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Implication

Phase 4 trial shows high 2-year survival after haploidentical stem cell transplantation in transfusion-dependent thalassaemia, highlighting an effective cure when matched donors are unavailable.

Key Points

To assess the efficacy and safety of haploidentical haematopoietic stem cell transplantation in patients with transfusion-dependent thalassaemia lacking fully matched donors.
Enrolled 134 patients with transfusion-dependent thalassaemia (median age 8.7 years; range 3–20 years) in an open-label, single-centre phase 4 trial with a median follow-up of 23.4 months.
Administered a uniform conditioning regimen of busulfan, cyclophosphamide, fludarabine, and anti-thymocyte globulin, alongside graft-versus-host disease prophylaxis comprising methotrexate, mycophenolate mofetil, and tacrolimus.
Achieved a 2-year overall survival of 93.4%, event-free survival of 92.6%, and graft-versus-host disease-free, relapse-free survival of 77.5%.
Observed cumulative incidence rates of 47.8% for all-grade acute graft-versus-host disease (aGVHD), 30.9% for grade 2–4 aGVHD, and 12.5% for grade 3–4 aGVHD.
Identified inferior overall survival associated with donor-specific antibodies (p=0.021), male-recipient/female-donor pairings (p=0.010), and hepatomegaly ≥5 cm (p=0.037).

Cite This Study

Lai et al. (2026) studied this question.

synapsesocial.com/papers/6a993586636c6408cfa7db73 https://doi.org/10.1111/bjh.70819

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Implication

Phase 4 trial shows high 2-year survival after haploidentical stem cell transplantation in transfusion-dependent thalassaemia, highlighting an effective cure when matched donors are unavailable.

Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase IV , open‐label, single‐centre trial

Discussion

Member takes

Implication

Key Points

Cite This Study

Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase IV , open‐label, single‐centre trial

Discussion

Member takes

Implication

Key Points

Cite This Study

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