Impact of Bone Marrow Transplant Vs. Supportive Care on Health Related Quality of Life in Patients with Severe Thalassemia in a Lower Middle-Income Country

Bone Marrow Transplantation (BMT) is the only established curative modality for severe hemoglobinopathies. Its positive impact on Health-Related Quality of Life (HRQoL) is well established in the West; however, its impact is less well documented and quantified in low- and middle-income countries. The aim of this study was to assess and compare HRQoL of patients with severe thalassemia (ST), i.e. having a thalassemia syndrome with inability to keep a spontaneous hemoglobin above 7 g/dL, at least 2 years from successful BMT to those on conventional supportive care. A comparative cross sectional study was conducted at the BMT Unit and Thalassemia Centre of the Childrenu0027s Hospital, Pakistan Institute of Medical Sciences in Islamabad, Pakistan. The study sample was selected through non probability purposive sampling. A total of 75 patients, largely coming from lower income strata and followed in a Government setting were included in the study. BMT was offered on a non-profit and subsidized basis so that financial limitations were not an absolute obstacle to access transplantation. Age-appropriate Child-self and parent-proxy reports of PedsQL 4.0 Generic core was used to evaluate HRQoL in 25 ex-thalassemic patients u003e 2 years after a successful matched-related BMT (group 1) and in 51 patients with ST on conventional supportive care (Group 2). Out of 76 patients enrolled in our study, Male/Female ratio was 50/26 with median age of 7 years (2-18years). Reliability analysis for checking agreement between child-self and parent-proxy report showed interclass correlation (ICC) of 0.878, with 95% Confidence interval of 0.805 to 0.925, thus showing good agreement between child-self and parent-proxy report. Statistical analysis of the data collected from both groups showed a significantly higher physical, emotional, social, school and overall functioning in ex-thalassemics after BMT compared to thalassemic patients on supportive care. We observed a very significant difference between group in physical, emotional, social and school functioning domain scores with means of 27.18 (p value 0.0001), 18.838 (p value 0.0001), 12.50 (p value 0.001) and 16.64 (p value 0.006) respectively. In conclusion, the impact of BMT in improving HRQoL of patients with severe thalassemia in all domains in a lower-income setting is very evident and possibly greater than in affluent countries .