Outcome of relapsed/refractory acute promyelocytic leukaemia in children, adolescents and young adult patients — a 25‐year Italian experience

Testi, Anna Maria; Mohamed, Sara; Diverio, Daniela; Piciocchi, Alfonso; Menna, Giuseppe; Rizzari, Carmelo; Timeus, Fabio; Micalizzi, Concetta; Nigro, Luca Lo; Santoro, Nicola; Masetti, Riccardo; Micheletti, Maria Vittoria; Ziino, Ottavio; Onofrillo, Daniela; Ladogana, Saverio; Putti, Caterina; Pierani, Paolo; Arena, Valentina; Zecca, Marco; Foà, Robin; Locatelli, Franco

doi:10.1111/bjh.17637

Cited by 4 publications

(4 citation statements)

References 19 publications

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“…Recently, a group from Italy reported relatively favorable outcomes (molecular CR rate of 96%, 10-y EFS rate of 67.7%, and 10-y OS rate of 79.4%) for 51 children with relapsed/refractory APL. 11 However, it should be noted that all these patients had received their frontline therapy with ATRA+chemotherapy without ATO. More recently, a single center from India reported similar outcomes (molecular CR rate of 94%, 5-y EFS rate of 67%, and 5-y OS rate of 73.6%) for 67 patients (including 13 patients 12 y old or younger) who had received upfront ATO-based therapy.…”

Section: Discussionmentioning

confidence: 99%

“…Reports on outcomes for children with relapsed/refractory APL are scarce. Recently, a group from Italy reported relatively favorable outcomes (molecular CR rate of 96%, 10-y EFS rate of 67.7%, and 10-y OS rate of 79.4%) for 51 children with relapsed/refractory APL 11. However, it should be noted that all these patients had received their frontline therapy with ATRA+chemotherapy without ATO.…”

Section: Discussionmentioning

confidence: 99%

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Gemtuzumab Ozogamicin Followed by Unrelated Cord Blood Transplantation With Reduced-intensity Conditioning for a Child With Refractory Acute Promyelocytic Leukemia

Yamada

Osumi

Kato

et al. 2022

Journal of Pediatric Hematology/Oncology

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There is no established treatment for patients with acute promyelocytic leukemia (APL) refractory to targeted therapies with all-trans retinoic acid (ATRA) and/or arsenic trioxide (ATO). We report here a case of an 8-month-old girl with APL who failed standard ATRA-combined chemotherapy. Although molecular remission was achieved after introducing ATRA/ATO combination therapy, molecular relapse occurred during the ATO consolidation courses. Subsequent molecular remission was rapidly achieved after administering 2 doses of gemtuzumab ozogamicin. She was successfully treated with unrelated cord blood transplantation using reduced-intensity conditioning. Gemtuzumab ozogamicin might be a preferable choice for patients with APL refractory to standard therapy.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Gemtuzumab Ozogamicin Followed by Unrelated Cord Blood Transplantation With Reduced-intensity Conditioning for a Child With Refractory Acute Promyelocytic Leukemia

Yamada

Osumi

Kato

et al. 2022

Journal of Pediatric Hematology/Oncology

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“…This data has been confirmed by the 25-year Italian experience on relapsed/refractory children and adolescents with APL. 27 Eight patients in CR2 were consolidated with auto-HSCT and 17 with allo-HSCT. No significant differences between the two transplant procedures have been observed; the 10-year OS and EFS were 87.5% vs 76% and 72.9% vs 70.6%, respectively ( Table 2 ).…”

Section: Acute Promyelocytic Leukemia (Apl)mentioning

confidence: 99%

Pediatric Autologous Hematopoietic Stem Cell Transplantation: Safety, Efficacy, and Patient Outcomes. Literature Review

Testi¹,

Moleti²,

Angi³

et al. 2023

PHMT

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Autologous stem cell transplantation (auto-HSCT) is a part of the therapeutic strategy for various oncohematological diseases. The auto-HSCT procedure enables hematological recovery after high-dose chemotherapy, otherwise not tolerable, by the infusion of autologous hematopoietic stem cells. Unlike allogeneic transplant (allo-HSCT), auto-HSCT has the advantage of lacking acute-graft-versus-host disease (GVHD) and prolonged immunosuppression, however, these advantages are counterbalanced by the absence of graft-versus-leukemia. Moreover, in hematological malignancies, the autologous hematopoietic stem cell source may be contaminated by neoplastic cells, leading to disease reappearance. In recent years, allogeneic transplant-related mortality (TRM) has progressively decreased, almost approaching auto-TRM, and many alternative donor sources are available for the majority of patients eligible for transplant procedures. In adults, the role of auto-HSCT compared to conventional chemotherapy (CT) in hematological malignancies has been well defined in many extended randomized trials; however, such trials are lacking in pediatric cohorts. Therefore, the role of auto-HSCT in pediatric oncohematology is limited, in both first- and second-line therapies and still remains to be defined. Nowadays, the accurate stratification in risk groups, according to the biological characteristics of the tumors and therapy response, and the introduction of new biological therapies, have to be taken into account in order to assign auto-HSCT a precise role in the therapeutic strategies, also considering that in the developmental age, auto-HSCT has a clear advantage over allo-HSCT, in terms of late sequelae, such as organ damage and second neoplasms. The purpose of this review is to report the results obtained with auto-HSCT in the different pediatric oncohematological diseases, focusing on the most significant literature data in the context of the various diseases and discussing this data in the light of the current therapeutic landscape.

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“…More recently, the 25-year Italian experience on the outcome of relapsed/refractory APL in children, adolescents and young adults has been reported. 28 Fifty-one patients (age < 18 years at initial diagnosis; median age 13.9 years at the time of the study), treated between May 1994 and May 2017 in 22 AIEOP (Associazione Italiana Ematologia e Oncologia Pediatrica) centers who experienced relapsed or refractory disease, were included in this study. All patients had received front-line ATRA and chemotherapy; salvage strategies were heterogeneous and, up to January 2008, were based on ATRA and intensive chemotherapy followed by allo- or auto-HSCT or different maintenance approaches for patients not eligible for transplant.…”

Section: Pediatric Experiencesmentioning

confidence: 99%

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

Testi¹

2022

Mediterr J Hematol Infect Dis

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The past three decades have brought major therapeutic advances in the treatment of 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 the achievement of 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 persistent molecular disease, allogeneic HSCT could be offered if a suitable donor is identified. Except for sporadic pediatric reports, most of the evidence for the use of HSCT for treatment of relapsed/refractory APL, comes from adult literature. We hereby provide a review of published pediatric data that evaluated the role of HSCT in children with refractory/recurrent APL disease. KEYWORDS: acute promyelocytic leukemia; relapse; hematopoietic stem cell transplant; children; adolescents

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Outcome of relapsed/refractory acute promyelocytic leukaemia in children, adolescents and young adult patients — a 25‐year Italian experience

Abstract: ting-oral-anticoagulants-doacs-increased-risk-of-recurrent-thrombotic-eve nts-in-patients-with-antiphospholipid-syndrome.

Cited by 4 publications

References 19 publications

Gemtuzumab Ozogamicin Followed by Unrelated Cord Blood Transplantation With Reduced-intensity Conditioning for a Child With Refractory Acute Promyelocytic Leukemia

Gemtuzumab Ozogamicin Followed by Unrelated Cord Blood Transplantation With Reduced-intensity Conditioning for a Child With Refractory Acute Promyelocytic Leukemia

Pediatric Autologous Hematopoietic Stem Cell Transplantation: Safety, Efficacy, and Patient Outcomes. Literature Review

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

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