# Unrelated Donor BMT vs. Immune Suppression for Newly Diagnosed Severe Aplastic Anemia in Children and Young Adults: BMT CTN Core Center Renewal for the Pediatric Blood and Marrow Transplant Consortium

> **NIH NIH UG1** · BOSTON CHILDREN'S HOSPITAL · 2020 · $187,669

## Abstract

Project Summary
Acquired severe aplastic anemia (SAA) is a rare bone marrow failure disorder with an
estimated annual incidence of 2 per million in North America (just over 600 new
diagnoses in the United States each year). The large majority of cases may be caused
by autoimmune destruction of hematopoietic stem cells (HSCs); accordingly SAA can be
treated and often cured by either immune suppression therapy (IST) or marrow
replacement through hematopoietic cell transplantation (HCT). HCT from a human
leukocyte antigen (HLA) matched sibling donor (MSD) is considered the standard for
initial therapy of younger, newly diagnosed patients with long-term survival rates of up to
95-100% in patients under 20. However, only 18-20% of patients will have matched
sibling donors, consequently, the large majority of patients receive IST for initial therapy.
We propose a phase II trial comparing up front matched unrelated donor (URD) HCT
with IST for children and very young adults with SAA. The primary endpointwould be
proportion of patients alive at 2 years and immune suppression free with adequate
counts. We have an open and accruing pilot study that is assessing key feasibility
issues with the clinical design including acceptance of randomization, timely acquisition
of donors, and safety of HCT. In addition, the pilot is testing the feasibility of acquiring,
shipping and processing key biological specimens obtained from patients located at
institutions across the country that will allow us in the future to elucidate the immunologic
phenotype and genomic landscape of the marrow, and to understand the mechanisms of
response and toxicity. Success of this pilot study and the phase III trial proposed in this
BMT CTN renewal application is strengthened by a joint effort between two strong,
established consortia: the North American Pediatric Aplastic Anemia Consortium
(NAPAAC) and the Pediatric Blood and Marrow Transplant Consortium (PBMTC). If
successful, this phase III trial will improve short-term immune suppression free and long-
term overall survival and quality of life in young patients with SAA.

## Key facts

- **NIH application ID:** 9931258
- **Project number:** 7UG1HL069254-20
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** Leslie S Kean
- **Activity code:** UG1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $187,669
- **Award type:** 7
- **Project period:** 2001-09-30 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9931258

## Citation

> US National Institutes of Health, RePORTER application 9931258, Unrelated Donor BMT vs. Immune Suppression for Newly Diagnosed Severe Aplastic Anemia in Children and Young Adults: BMT CTN Core Center Renewal for the Pediatric Blood and Marrow Transplant Consortium (7UG1HL069254-20). Retrieved via AI Analytics 2026-06-10 from https://api.ai-analytics.org/grant/nih/9931258. Licensed CC0.

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