Summary:
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.
Similar content being viewed by others
References
Raimondi SC, Chang MN, Ravindranath Y et al. Chromosomal abnormalities in 478 children with acute myeloid leukemia: clinical characteristics and treatment outcome in a cooperative pediatric oncology group study-POG 8821. Blood 1999; 94: 3707–3716.
Stevens RF, Hann IM, Wheatley K, Gray RG . Marked improvements in outcome with chemotherapy alone in paediatric acute myeloid leukemia: results of the United Kingdom Medical Research Council's 10th AML trial. MRC Childhood Leukaemia Working Party. Br J Haematol 1998; 101: 130–140.
Woods WG, Neudorf S, Gold S et al. A comparison of allogeneic bone marrow transplantation, autologous bone marrow transplantation, and aggressive chemotherapy in children with acute myeloid leukemia in remission. Blood 2001; 97: 56–62.
Vardiman JW, Harris NL, Brunning RD . The World Health Organization (WHO) classification of the myeloid neoplasms. Blood 2002; 100: 2292–2302.
Fenaux P, Chastang C, Chevret S et al. A randomized comparison of all transretinoic acid (ATRA) followed by chemotherapy and ATRA plus chemotherapy and the role of maintenance therapy in newly diagnosed acute promyelocytic leukemia. The European APL Group. Blood 1999; 94: 1192–1200.
Sanz MA, Martin G, Gonzalez M et al. Risk-adapted treatment of acute promyelocytic leukemia with all-trans-retinoic acid and anthracycline monochemotherapy: a multicenter study by the PETHEMA group. Blood 2004; 103: 1237–1243.
Meloni G, Diverio D, Vignetti M et al. Autologous bone marrow transplantation for acute promyelocytic leukemia in second remission: prognostic relevance of pretransplant minimal residual disease assessment by reverse-transcription polymerase chain reaction of the PML/RAR alpha fusion gene. Blood 1997; 90: 1321–1325.
Sanz M, Arcese W, De la Rubia J et al. Stem cell transplantation (SCT) for acute promyelocytic leukaemia in the ATRA era: a survey of the European Blood and Marrow Transplantation Group (EBMT). Blood 2000; 96 (Suppl. 1): 522a.
Thomas X, Dombret H, Cordonnier C et al. Treatment of relapsing acute promyelocytic leukemia by all-trans retinoic acid therapy followed by timed sequential chemotherapy and stem cell transplantation. APL Study Group. Leukemia 2000; 14: 1006–1013.
Soignet SL, Frankel SR, Douer D et al. United States multicenter study of arsenic trioxide in relapsed acute promyelocytic leukemia. J Clin Oncol 2001; 19: 3852–3860.
Lazo G, Kantarjian H, Estey E et al. Use of arsenic trioxide (As2O3) in the treatment of patients with acute promyelocytic leukemia: the M.D. Anderson experience. Cancer 2003; 97: 2218–2224.
Lo-Coco F, Romano A, Mengarelli A et al. Allogeneic stem cell transplantation for advanced acute promyelocytic leukemia: results in patients treated in second molecular remission or with molecularly persistent disease. Leukemia 2003; 17: 1930–1933.
Storb R, Deeg HJ, Whitehead J et al. Methotrexate and cyclosporine compared with cyclosporine alone for prophylaxis of acute graft versus host disease after marrow transplantation for leukemia. N Engl J Med 1986; 314: 729–735.
Chao NJ, Schmidt GM, Niland JC et al. Cyclosporine, methotrexate, and prednisone compared with cyclosporine and prednisone for prophylaxis of acute graft-versus-host disease. N Engl J Med 1993; 329: 1225–1230.
Meyers JD, Reed EC, Shepp DH et al. Acyclovir for prevention of cytomegalovirus infection and disease after allogeneic marrow transplantation. N Engl J Med 1988; 318: 70–75.
Shulman HM, Sullivan KM, Weiden PL et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med 1980; 69: 204–217.
Rowlings PA, Przepiorka D, Klein JP et al. IBMTR Severity Index for grading acute graft-versus-host disease: retrospective comparison with Glucksberg grade. Br J Haematol 1997; 97: 855–864.
Lansky SB, List MA, Lansky LL et al. The measurement of performance in childhood cancer patients. Cancer 1987; 60: 1651–1656.
Acknowledgements
JPB is a recipient of a Fellowship Grant from the Lady Tata Foundation. The study was supported by the Frank J Hanna Jr, Research Fund.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
Bourquin, J., Thornley, I., Neuberg, D. et al. Favorable outcome of allogeneic hematopoietic stem cell transplantation for relapsed or refractory acute promyelocytic leukemia in childhood. Bone Marrow Transplant 34, 795–798 (2004). https://doi.org/10.1038/sj.bmt.1704676
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/sj.bmt.1704676
- Springer Nature Limited