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

A Study of Clinical Profile and Outcome in Acute Viral Hepatitis E.

Introduction: Hepatitis E is the most common cause of acute viral hepatitis in the adult population in India. Acute viral hepatitis E (HEV) is generally mild and self-limiting and resolves within six weeks, with no chronic sequelae. Pregnant women are at increased risk with the risk increasing as the pregnancy progresses near term. Methods: We performed a prospective study of 100 patients who presented to Dept. of Medicine, Civil Hospital, Ahmedabad with jaundice (IgM anti-HEV positive) during 2011-2012 and data were analyzed for the clinical profile and outcome of HEV. Results: The most common age group affected was 21-30 years with overall M:F ratio of 3.4:1. Most common presenting symptom was jaundice (100%) followed by nausea/ vomiting (87%). On presentation, serum bilirubin level was elevated in 89% patients with SGPT raised in 86%. PT was prolonged in 39% patients. Anemia (Hb < 7) was seen in 17% while 9% of patients had renal involvement. Hepatic encephalopathy was seen in 15%. In this study, 12 women were pregnant in whom IUD occurred in 66% with maternal mortality in 33%. Overall mortality was 7%. Conclusion: We found that majority of cases were cured with supportive treatment while risk of mortality and morbidity was more in pregnant women.