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Haematologica. 2019 Apr 4. pii: haematol.2018.206540. doi: 10.3324/haematol.2018.206540. [Epub ahead of print]

Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.

Author information

1
Pediatric Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, TX.
2
Children's Hospital Colorado.
3
Children's Hospital of Philadelphia, Philadelphia, PA.
4
Baylor College of Medicine, Houston TX.
5
Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN.
6
Hackensack University Medical Center, Hackensack, NJ.
7
Children's Hospital Los Angeles, Los Angeles CA.
8
Brown University, Providence RI.
9
University of Florida, Gainesville FL.
10
Oregon Health & Science University, Portland OR.
11
Johns Hopkins University, Baltimore MD.
12
Emory University, Atlanta GA.
13
Nationwide Childrens Hospital, Columbus.
14
Seattle Children's Hospital, Seattle, WA.
15
Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ.
16
Indiana University School of Medicine, Indianapolis, IN.
17
Duke, Durham NC.
18
UCSF Benioff Children's Hospital, San Francisco.
19
Yale, New Haven CT.
20
SickKids Hospital, Toronto, ON, CAN.
21
Stanford University School of Medicine, Palo Alto, CA.
22
University of Michigan, Ann Arbor, MI.
23
Lurie Children's Hospital, Chicago IL.
24
Cleveland Clinic, Cleveland OH.
25
Hofstra Northwell School of Medicine, Hempstead, NY.
26
Institutional Centers for Clinical and Translational Research, Boston Children Hospital, Boston MA.
27
Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston MA.
28
Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston MA akiko.shimamura@childrens.harvard.edu.

Abstract

Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia are also limited for pediatric patients. The clinical features and outcomes for 314 children treated from 2002-2014 with immunosuppressive therapy for acquired severe aplastic anemia were analyzed retrospectively from 25 institutions in the North American Pediatric Aplastic Anemia Consortium. The majority of subjects (n=264) received horse anti-thymocyte globulin plus cyclosporine with 61 months median follow up. Following horse anti-thymocyte globulin/cyclosporine, 71.2% (95% CI: 65.3,76.6) achieved an objective response. In contrast to adult studies, the quality of response attained in pediatric patients was high, with 59.8% (95% CI: 53.7,65.8) reaching a complete response and 68.2% (95% CI: 62.2,73.8) attaining at least a very good partial response with a platelet count ≥50,000/μL. At 5 years post-horse anti-thymocyte globulin/cyclosporine, overall survival was 93% (95% CI: 89,96), but event free survival without subsequent treatment was only 64% (95% CI: 57,69) without a plateau. Twelve of 171 evaluable patients (7%) acquired clonal abnormalities after diagnosis with a median time of 25.2 months (range 4.3-71 months) post-treatment. Myelodysplastic syndrome or leukemia developed in 6/314 (1.9%). For relapsed/refractory disease, treatment with a hematopoietic stem cell transplant had a superior event-free survival compared to second immunosuppressive therapy treatment in a multivariate analysis (HR=0.19, 95% CI:0.08, 0.47, p=0.0003). This study highlights the need for improved therapies leading to sustained high quality remission for children with severe aplastic anemia.

KEYWORDS:

Bone Marrow Failure; Pediatric aplastic anemia; Stem Cell Transplantation

PMID:
30948484
DOI:
10.3324/haematol.2018.206540
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