1. ¹Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA
2. ²Division of Hematology/Oncology, Children's Hospital Boston, Harvard Medical School, Boston, MA, USA
3. ³Department of Biostatistical Science, Dana-Farber Cancer Institute, Boston, MA, USA

Correspondence: Dr E Guinan, Department of Pediatric Oncology, Dana-Farber Cancer Institute, 44 Binney Street, Boston, MA 02115, USA. E-mail: Eva_Guinan@dfci.harvard.edu

Received 30 April 2004; Accepted 21 June 2004; Published online 6 September 2004.

Abstract

The optimal therapy for children with relapsed or refractory acute promyelocytic leukemia (APL) is unclear. We therefore reviewed our institutional outcomes for children undergoing allogeneic hematopoietic stem cell transplantation (HSCT) for advanced APL. Between 1986 and 2003, 12 allogeneic HSCTs (five related donor, seven unrelated donor) were performed for 11 patients (median age, 13 years) with relapsed (n=8) or refractory (n=3) APL. All patients engrafted, after a median of 18.5 days. Grade B–D acute graft-versus-host disease (GVHD) developed after five transplants (42%; 90% CI, 18–68%), and the cumulative incidence of chronic GVHD was 45% (90% CI, 19–71%). The cumulative incidence of overt relapse post-HSCT was 10% (90% CI, 0–28%). The overall 5-year survival was 73% (90% confidence interval (CI), 51–95%), with a median post-HSCT follow-up of 64 months. The Lansky/Karnofsky performance scores are 100% in six of eight survivors. In view of the low risk of subsequent relapse and favorable survival suggested by other reports and our own experience, we continue to recommend allogeneic HSCT for children with advanced APL for whom a suitably HLA-matched donor is identified.