Affordable Access

deepdyve-link
Publisher Website

Treatment-related toxicities in children with acute lymphoblastic leukaemia predisposition syndromes.

Authors
  • Schmiegelow, Kjeld1
  • 1 Department of Pediatrics and Adolescent Medicine, University Hospital Rigshospitalet, Copenhagen, Denmark; Institute of Clinical Medicine, University of Copenhagen, Denmark; Division of Pediatric Hematology/Oncology, New York, USA; Perlmutter Cancer Center, NYU Langone Medical Center, New York, USA. Electronic address: [email protected]
Type
Published Article
Journal
European journal of medical genetics
Publication Date
December 2016
Volume
59
Issue
12
Pages
654–660
Identifiers
DOI: 10.1016/j.ejmg.2016.02.006
PMID: 26876989
Source
Medline
Keywords
License
Unknown

Abstract

Although most children with acute lymphoblastic leukaemia (ALL) do not harbor germline mutations that strongly predispose them to development of this malignancy, large syndrome registries and detailed mapping of exomes or whole genomes of familial leukaemia kindreds have revealed that 3-5% of all childhood ALL cases are due to such germline mutations, but the figure may be higher. Most of these syndromes are primarily characterized by their non-malignant phenotype, whereas ALL may be the dominating or even only striking manifestation of the syndrome in some families. Identification of such ALL patients is important in order to adjust therapy and offer genetic counseling and cancer surveillance to mutation carriers in the family. In the coming years large genomic screening projects are expected to reveal further hitherto unrecognised familial ALL syndromes. The treatment of ALL cases harboring cancer predisposing mutations can be challenging for both the physician and the patient due to their preexisting symptoms, their reduced tolerance to radio- and/or chemotherapy with enhanced risk of life-threatening organ toxicities, and the paucity of data from ALL patients with the same or similar syndromes being treated by contemporary protocols. Recent studies clearly indicate that many of these patients stand a good chance of cure, and that they should be offered chemotherapy with the intention to cure. Some of these syndromes are characterized by reduced tolerance to radiotherapy and/or specific anticancer agents, while others are not. This review summarises our current knowledge on the risk of acute toxicities for these ALL patients and provides guidance for treatment adjustments.

Report this publication

Statistics

Seen <100 times