Outcome of Pediatric Acquired Aplastic Anemia: A Developing World Experience. (1st February 2014)

• Record Type: Journal Article
• Authors: Ramzan, Mohammed; Yadav, S.P.; Zafar, Mir Sadaqat Hassan; Dinand, Veronique; Sachdeva, Anupam
• Is Part Of: Pediatric hematology and oncology. Volume 31:Number 1(2014:Feb.)
• Page Start: 29
• Page End: 38
• Publication Date: 2014-02-01
• Subjects: Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Hematologic Diseases -- Child; Hematologic Diseases -- Infant; Neoplasms -- Child; 618.9215
• DOI: 10.3109/08880018.2013.807898
• Languages: English
• ISSNs: 0888-0018

Abstract

<abstract> <title> <x xml:space="preserve">Abstract</x> </title> <p> <italic>Introduction:</italic> Outcome data of children with acquired aplastic anemia (AA) are lacking from the developing world. Here, we describe the same from a centre in North India. <italic>Methods</italic>: Retrospective data regarding medical history, physical examination, complete blood count, bone marrow aspirate, and biopsy were retrieved for all children <18 years, with acquired AA admitted between January 2005 and June 2012. In addition, the outcome data after immunosuppressive therapy (IST) or bone marrow transplant (BMT) was obtained. <italic>Results</italic>: A total of 61 children were diagnosed with AA (Inherited-18 and acquired-43). Among 43 children with acquired AA, 3 had nonsevere and 40 had severe. One patient with nonsevere AA died of sepsis and 2 recovered spontaneously. Of the 40 remaining children with severe AA, 10 refused therapy and 3 died due to severe sepsis prior to any therapy. Five underwent upfront matched sibling donor BMT and one post-IST failure. Four year overall survival (OS) and event free survival (EFS) for children undergoing BMT was 100% and 80 ± 17.9, respectively. Out of 22 treated with IST, 20 were evaluable for response. Seventeen received one course and 3 received two course of IST. The overall response to IST was seen in 14/20 (70%). Only two achieved complete response while remaining 12 had partial response. The 4-year estimated OS and EFS for children treated with IST was 74.4 ± 12.1% and 65.6 ± 12.2. <italic>Conclusion</italic>: Outcomes for children with AA are encouraging in the developing world although barriers like sepsis and treatment abandonment remain. BMT offers faster and complete recovery.</p> </abstract>