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<p>Hematopoietic stem cell transplantation (HSCT) is the only definitive curative treatment&#xa0;available for transfusion-dependent thalassemia (TDT). However, the availability of full&#xa0;matched donors remains a limiting factor. We aim to evaluate the outcomes of&#xa0;haploidentical HSCT in children with TDT at a single center in South India.&#xa0;This retrospective observational study analyzed 33 children with TDT who underwent T-cell replete haploidentical HSCT between 2021 and 2025. Pre-transplant risk was&#xa0;assessed using Pesaro criteria. All patients received pre-HSCT immunosuppressive&#xa0;chemotherapy, desensitization (if donor-specific anti-HLA antibodies (DSA) positive),&#xa0;and G-CSF plus plerixafor mobilized peripheral blood stem cells. Conditioning&#xa0;regimens were tailored, and GVHD prophylaxis were post-transplant&#xa0;cyclophosphamide (PTCy) based.&#xa0;Sustained engraftment occurred in 82% (27/33) of patients. DSA positivity (27%),&#xa0;especially with MFI &gt;10,000 was associated with graft failure. GVHD occurred in 36%&#xa0;of patients; severe GVHD contributed to two deaths. The overall survival was 67%,&#xa0;with Pesaro class-specific survival of 100% (Class 1), 78% (Class 2), and 50% (Class&#xa0;3). The median duration of follow-up was 22 months (3 months to 57 months).&#xa0;PTCy based haploidentical HSCT is a viable curative strategy for TDT, especially in&#xa0;resource-constrained settings, when full matched donors are unavailable. Favourable&#xa0;outcomes in Pesaro Class 1 and 2, on par with matched donor HSCT, highlight the&#xa0;importance of early HSCT before development of iron overload.</p>

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Outcomes of Haploidentical Hematopoietic Stem Cell Transplantation in Transfusion-Dependent Thalassemia: A Single-Center Experience from South India

  • 期刊论文

  • 发表日期：

    2026年1月7日

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  • Vimal Kumar,
  • Rishab Bharadwaj,
  • Deenadayalan Munirathnam

摘要

Hematopoietic stem cell transplantation (HSCT) is the only definitive curative treatment available for transfusion-dependent thalassemia (TDT). However, the availability of full matched donors remains a limiting factor. We aim to evaluate the outcomes of haploidentical HSCT in children with TDT at a single center in South India. This retrospective observational study analyzed 33 children with TDT who underwent T-cell replete haploidentical HSCT between 2021 and 2025. Pre-transplant risk was assessed using Pesaro criteria. All patients received pre-HSCT immunosuppressive chemotherapy, desensitization (if donor-specific anti-HLA antibodies (DSA) positive), and G-CSF plus plerixafor mobilized peripheral blood stem cells. Conditioning regimens were tailored, and GVHD prophylaxis were post-transplant cyclophosphamide (PTCy) based. Sustained engraftment occurred in 82% (27/33) of patients. DSA positivity (27%), especially with MFI >10,000 was associated with graft failure. GVHD occurred in 36% of patients; severe GVHD contributed to two deaths. The overall survival was 67%, with Pesaro class-specific survival of 100% (Class 1), 78% (Class 2), and 50% (Class 3). The median duration of follow-up was 22 months (3 months to 57 months). PTCy based haploidentical HSCT is a viable curative strategy for TDT, especially in resource-constrained settings, when full matched donors are unavailable. Favourable outcomes in Pesaro Class 1 and 2, on par with matched donor HSCT, highlight the importance of early HSCT before development of iron overload.