HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?

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...

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Main Author: Anna Maria Testi
Format: Article
Language:English
Published: PAGEPress Publications 2022-04-01
Series:Mediterranean Journal of Hematology and Infectious Diseases
Online Access:https://www.mjhid.org/index.php/mjhid/article/view/4931
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author Anna Maria Testi
author_facet Anna Maria Testi
author_sort Anna Maria Testi
collection DOAJ
description 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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spelling doaj-art-9734f249560642fe9bec07a492b9cf3f2024-12-02T07:14:35ZengPAGEPress PublicationsMediterranean Journal of Hematology and Infectious Diseases2035-30062022-04-0114110.4084/MJHID.2022.038HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?Anna Maria Testi01Department of Cellular Biotechnologies and Hematology, University La Sapienza , Rome 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 https://www.mjhid.org/index.php/mjhid/article/view/4931
spellingShingle Anna Maria Testi
HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?
Mediterranean Journal of Hematology and Infectious Diseases
title HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?
title_full HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?
title_fullStr HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?
title_full_unstemmed HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?
title_short HAS HEMATOPOIETIC STEM CELL TRANSPLANTATION A ROLE IN THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA?
title_sort has hematopoietic stem cell transplantation a role in the treatment of children and adolescents with acute promyelocytic leukemia
url https://www.mjhid.org/index.php/mjhid/article/view/4931
work_keys_str_mv AT annamariatesti hashematopoieticstemcelltransplantationaroleinthetreatmentofchildrenandadolescentswithacutepromyelocyticleukemia