Quality Matters – Hematopoietic Stem Cell Transplantation versus Transfusion and Chelation in Thalassemia Major

Objective: To compare quality of life of children with thalassemia major who haveundergone stem cell transplantation with those on regular transfusion. Methods: Thestudy included 40 children who underwent transplantation and 40 children and 20 adults onregular transfusion and iron chelation therapy. The quality of life assessment was doneusing the Pediatric Quality of Life Inventory 4.0 Generic Core Scale. Results: The meantotal summary score, psychosocial summary score and physical score was 92, 91 and92.8, respectively in transplant group and 83, 82.7 and 83.6, respectively in children intransfusion group. The adult group on transfusion showed overall poorer scores of 74.9, 76and 73.9, respectively. The average scores in all domains were significantly (P<0.05) lowerand drop steeply in second decade in transfusion group. Conclusion: Allogeneic stem celltransplantation improves quality of life in thalassemia major.

Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency Disorders: Experience from a Referral Center in India

Objective: To share experience of over 15 years in hematopoieticstem cell transplantation in children with primaryimmunodeficiency disorders.Design: Medical record review.Setting: A referral center for pediatric hemato-oncologicaldisorders.Participants: Children (<18 y) diagnosed to have primaryimmune deficiencies who underwent hematopoietic stem celltransplantation between 2002 and August 2017.Main outcome measures: Disease-free survival, morbidity andmortality.Results: 85 primary immunodeficiency disorder transplants wereperformed with engraftment noted in 80 (94%) transplants and anoverall survival of 67%. The conditioning regimen wasindividualized based on the underlying immune defect. Mixedchimerism was noted in 20% children with 56% (9/16) remainingdisease-free. Graft versus host disease was noted in 33 (39.2%)children with most seen in children with chronic granulomatousdisease. Severe combined immune deficiency transplants weremainly complicated by infections. Immune cytopeniascomplicated Wiskott Aldrich syndrome and Hemophagocyticlymphohistiocytosis transplants. 29.4% (25/85) childrenunderwent haploidentical transplant in our cohort with a survival of70% in this group. Infectious complications were the mostcommon cause of death.Conclusion: Primary immunodeficiency disorders are curable inIndia when transplanted in centers with experienced and trainedpediatric transplant physicians and intensivists