DefinePK

Abstract

Background: Standard first-line treatment for newly diagnosed severe aplastic anemia patients is allogeneic hematopoietic stem cell transplant in the presence of a suitable donor and horse anti-thymocyte globulin, ciclosporin and eltrombopag for patients lacking a donor.


Objectives: The aim of our study is to look at the quality of life in patients who received allogeneic hematopoietic stem cell transplant from matched related donors (MRD) in a low-middle income country.


Study Design: This single-center retrospective study conducted at Clinical Hematology and Bone Marrow Transplant Department, National University of Medical Sciences, Rawalpindi, Pakistan included 68 patients who received MRD allogeneic hematopoietic stem cell transplant between 2015 and 2024. WHOQOL-BREF was used to assess quality of life. Statistical analysis was done using SPSS version 27.


Results: Median age at the time of transplant was 21.5 (range: 9-48) years consisting of 57 (83.8%) males and 11 (16.2%) females. Median CD34? stem cell dose infused was 4.98 (range: 1.53–14.58) × 10? cells/kg. Neutrophil and platelet engraftment were achieved on median of day +13 (range: 9–25) and day +22 (range: 14–71) respectively. Most frequent complications included cytomegalovirus reactivation in 21 patients (30.9%) and graft-versus-host disease (Grade II-IV/Extensive) in 19 patients (27.9 %). Median duration from transplant to day of assessment of quality of life was 2573 (range: 386-4992) days. Mean quality of life scores were 13 (±18.42) in physical, 82.04 (±19.65) in psychological, 75.73 (±18.36) in social relations and 75.14 (±17.71) in environmental domains. Increasing recipient age showed a negative correlation with quality of life scores in physical (r = -0.2, p < 0.05), psychological (r = -0.1) and environmental (r = -0.2) domains. Increasing donor age had negative impact on recipient’s quality of life in all domains while statistical significance was seen only in environmental domain (r = -0.2, p < 0.05). Male recipients reported significantly higher psychological (p = 0.01) and environmental (p = 0.01) scores, with better scores in physical (p = 0.12) and social relations (p = 0.37) domains. Recipients who received bone marrow harvest alone exhibited superior quality of life scores in physical (p=0.44), psychological (p=0.93) and social relations (p = 0.02) compared to peripheral blood stem cells or a combination of both. Chronic graft-versus-host disease had significant adverse impact on quality of life in physical (p = 0.07) domain with lower scores in psychological (p = 0.48), social relations (p = 0.33) and environmental (p = 0.0.34) domains as well. Patients with cytomegalovirus reactivation had significantly worse psychological scores (p = 0.03) and physical (p = 0.06) and environmental (p = 0.06) scores.
Conclusion: Allogeneic hematopoietic stem cell transplant is curative therapeutic option for aplastic anemia and patients report good long-term quality of life. Younger recipient age, male gender and bone marrow harvest as the stem cell source were associated with better quality of life. Chronic graft versus host disease is a major detriment to quality of life, underscoring the need for chronic GVHD prevention and management strategies.