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<p>Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for aplastic anemia (AA), especially in younger patients with matched sibling donors (MSD). However, in low-resource settings, delayed access to HSCT and pre-transplant complications can adversely impact outcomes. This study evaluates long-term survival and key prognostic factors in AA patients undergoing HSCT at a single tertiary center in North India. A total of 54 patients who underwent hematopoietic stem cell transplantation (HSCT) between 2007 and 2024 for either immune-mediated or inherited aplastic anemia were retrospectively analyzed. Of these, 90.7% received transplants from matched sibling donors (MSD), with peripheral blood stem cells (PBSCs) used as the graft source in 94.5% of cases. Outcomes were assessed using Kaplan-Meier survival analysis. Cox regression was used to identify predictors of overall survival (OS) and GVHD and relapse-free survival (GRFS). The median age was 20 years (Range: 8–44 years), and 74% of patients were male. Immune-mediated AA accounted for 83% of cases. Median time from diagnosis to transplant was 8 months. Acute GVHD occurred in 27.6%, while chronic GVHD developed in 35.2%. Five-year OS and GRFS were 78.5% and 61.3%, respectively. Multivariable analysis showed that higher number of pre-HSCT PRBC transfusions and older donor age predicted inferior GRFS. For OS, older donor age and grade ≥ 2 mucositis were independent adverse predictors. Despite resource limitations, HSCT outcomes for AA in this cohort were comparable to global data. Early referral, infection control, and minimization of transfusion burden are critical to improving outcomes. Donor age and the occurrence of grade ≥ 2 mucositis emerged as significant prognostic indicators for survival, underscoring the critical role of donor selection and optimized supportive care in the transplant setting.</p>

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Donor Age and Pre-Transplant Transfusion Burden Independently Predict GVHD and Relapse-Free Survival (GRFS) in Aplastic Anemia

  • 期刊论文

  • 发表日期：

    2026年1月4日

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  • Sarthak Wadhera,
  • Rudra Narayan Swain,
  • Aarushi Sahni,
  • Charanpreet Singh,
  • Aditya Jandial,
  • Arihant Jain,
  • Man Updesh Sachdeva,
  • Rekha Hans,
  • Prateek Bhatia,
  • Deepesh Lad,
  • Gaurav Prakash,
  • Deepak Bansal,
  • Pankaj Malhotra,
  • Alka Khadwal

摘要

Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for aplastic anemia (AA), especially in younger patients with matched sibling donors (MSD). However, in low-resource settings, delayed access to HSCT and pre-transplant complications can adversely impact outcomes. This study evaluates long-term survival and key prognostic factors in AA patients undergoing HSCT at a single tertiary center in North India. A total of 54 patients who underwent hematopoietic stem cell transplantation (HSCT) between 2007 and 2024 for either immune-mediated or inherited aplastic anemia were retrospectively analyzed. Of these, 90.7% received transplants from matched sibling donors (MSD), with peripheral blood stem cells (PBSCs) used as the graft source in 94.5% of cases. Outcomes were assessed using Kaplan-Meier survival analysis. Cox regression was used to identify predictors of overall survival (OS) and GVHD and relapse-free survival (GRFS). The median age was 20 years (Range: 8–44 years), and 74% of patients were male. Immune-mediated AA accounted for 83% of cases. Median time from diagnosis to transplant was 8 months. Acute GVHD occurred in 27.6%, while chronic GVHD developed in 35.2%. Five-year OS and GRFS were 78.5% and 61.3%, respectively. Multivariable analysis showed that higher number of pre-HSCT PRBC transfusions and older donor age predicted inferior GRFS. For OS, older donor age and grade ≥ 2 mucositis were independent adverse predictors. Despite resource limitations, HSCT outcomes for AA in this cohort were comparable to global data. Early referral, infection control, and minimization of transfusion burden are critical to improving outcomes. Donor age and the occurrence of grade ≥ 2 mucositis emerged as significant prognostic indicators for survival, underscoring the critical role of donor selection and optimized supportive care in the transplant setting.