Study of the Outcome of Pediatric Patients with Severe Aplastic Anemia treated with Immunosuppressive Therapy at Ain Shams University Pediatric Hospital

Abstract

Aplastic anemia (AA) is life threatening disorder in pediatric age group with an increasing incidence nowadays. Hematopoietic stem cell transplant being the 1st line therapy; immunosuppressive therapy (IST) is the alternative therapy and is the most commonly used modality of treatment especially in the developing countries. Aim of the Work: to assess the outcome of IST in children with severe and very severe AA. Patients and Methods: Data for 23 children treated with IST from January 2010 to January 2019 (10 years) were retrieved from clinic records. IST included rabbit anti thymocyte globulin (ATG) along with sandimmune and another group were treated by sandimmune alone. Results: Patient characteristics included median age (9) with 73.9% male and 26.1% female. With median interval between diagnosis and start of IST 2(1 -5) months and around 30.4% with hepatitis A associated aplastic anemia. Complete response, and no response were seen in 4 (50%) patients and 4(50%) patients, respectively in patients received ATG and sandimune. While in patients received sandimune alone complete response, partial response and no response were seen in 7(46.7%) patients, 5 (33.3%) patients, and 3 (20%) patients respectively. The median time to best response in the whole cohort was 2 months. There was no difference in outcome related to severity of AA, or higher Hb or platelet level. There was a significantly better rate of response in both groups of patients with higher initial TLC count (p=0.001) initial ANC (p=0.002), initial ALC (p=0.001), and initial ARC (p=0.014) before start of IST. An overall response rate in both groups around of 43.47% reported a 5-year OS. With 45.5 of patients with complete response had HAAA. A delayed time to complete response with prolonged requirement of cyclosporine therapy was detected in the study. Conclusion: In a developing country setting, IST with ATG and cyclosporine seems to be an alternative treatment for children with aplastic anemia lacking MRD.