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Review
. 2022 May 1;14(1):e2022038.
doi: 10.4084/MJHID.2022.038. eCollection 2022.

Has Hematopoietic Stem Cell Transplantation a Role in the Treatment of Children and Adolescents with Acute Promyelocytic Leukemia?

Anna Maria Testi  1 , Paolo Musiu  1 , Maria Luisa Moleti  1 , Saveria Capria  1 , Walter Barberi  1
Affiliations
Review

Has Hematopoietic Stem Cell Transplantation a Role in the Treatment of Children and Adolescents with Acute Promyelocytic Leukemia?

Anna Maria Testi et al. Mediterr J Hematol Infect Dis. .

Abstract

The past three decades have brought major therapeutic advances in treating acute promyelocytic leukemia (APL) both in adults and children. The current state-of-the-art treatment with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) in combination or not with chemotherapy results in long-lasting remission and cure in more than 90% of newly diagnosed patients. These treatments have made relapse a rare event. The detection of PML-RARA transcript by polymerase chain reaction (PCR) during treatment and follow-up can predict a hematological relapse. All studies have suggested a survival benefit in patients with molecular relapse given pre-emptive therapy compared with those treated at the time of overt hematological relapse. ATO-based regimens seem to be effective for achieving a second molecular complete remission (CR). Patients in second molecular CR are generally considered candidates for autologous hematopoietic stem cell transplant (HSCT), while for those with a persistent molecular disease, allogeneic HSCT should be offered if a suitable donor is identified. Except for sporadic pediatric reports, most of the evidence for using HSCT to treat relapsed/refractory APL comes from adult literature. Therefore, we now provide a review of published pediatric data that evaluated the role of HSCT in children with refractory/recurrent APL disease.

Keywords: Acute promyelocytic leukemia; Adolescents; Children; Hematopoietic stem cell transplant; Relapse.

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Conflict of interest statement

Competing interests: The authors declare no conflict of Interest.

Figures

Figure 1
Figure 1
ECOG E2491 and CALGB C9710 trials. Event-free survival following hematopoietic stem cell transplant for childhood relapsed/refractory acute promyelocytic leukemia.
Figure 2
Figure 2
Center for International Blood and Marrow Transplantation Research (CIBMTR). Treatment-related mortality in patients undergoing allogeneic and autologous transplant for acute promyelocytic leukemia in second complete remission.
Figure 3
Figure 3
Center for International Blood and Marrow Transplantation Research (CIBMTR). Overall survival in patients undergoing allogeneic and autologous transplant for acute promyelocytic leukemia in second complete remission.
Figure 4
Figure 4
Overall survival for Italian children/adolescents undergoing allogeneic- or autologous-hematopoietic stem cell transplant in second complete remission.
Figure 5
Figure 5
Event-free survival for Italian children/adolescents undergoing allogeneic- or autologous-hematopoietic stem cell transplant in second complete remission.
Figure 6
Figure 6
C) Overall survival, D) Event-free survival of adult and pediatric patients with acute promyelocytic leukemia in second complete remission consolidated with autologous-stem cell transplant versus arsenic trioxide-based maintenance regimen.
Figure 7
Figure 7
Treatment algorithm for relapsed pediatric APL. *TTR=time to relapse; **mos=months; ~ATO=arsenic trioxide; ~~ATRA=all-trans retinoic acid; ^mCR=molecular complete remission; ^^GO=gentuzumab ozogamicin; °CT=chemotherapy; °°HSC=hematopoietic stem cells; ˤHSCT=hematopoietic stem cell transplant.
See this image and copyright information in PMC

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