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Pediatr Hematol Oncol. 2014 Feb;31(1):29-38. doi: 10.3109/08880018.2013.807898. Epub 2013 Jun 26.

Outcome of pediatric acquired aplastic anemia: a developing world experience.

Author information

1
Pediatric Hematology Oncology & BMT Unit, Department of Pediatrics, Sir Ganga Ram Hospital , New Delhi , India.

Abstract

INTRODUCTION:

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.

METHODS:

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.

RESULTS:

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.

CONCLUSION:

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.

PMID:
23802844
DOI:
10.3109/08880018.2013.807898
[Indexed for MEDLINE]

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