Recommendations for surveillance for children with leukemia-predisposing conditions

dc.contributor.authorPorter, C.C.
dc.contributor.authorDruley, T.E.
dc.contributor.authorErez, A.
dc.contributor.authorKuiper, R.P.
dc.contributor.authorOnel, K.
dc.contributor.authorSchiffman, J.D.
dc.contributor.authorSchneider, K.W.
dc.contributor.authorScollon, S.R.
dc.contributor.authorScott, H.S.
dc.contributor.authorStrong, L.C.
dc.contributor.authorWalsh, M.F.
dc.contributor.authorNichols, K.E.
dc.date.issued2017
dc.description.abstractLeukemia, the most common childhood cancer, has long been recognized to occasionally run in families. The first clues about the genetic mechanisms underlying familial leukemia emerged in 1990 when Li-Fraumeni syndrome was linked to TP53 mutations. Since this discovery, many other genes associated with hereditary predisposition to leukemia have been identified. Although several of these disorders also predispose individuals to solid tumors, certain conditions exist in which individuals are specifically at increased risk to develop myelodysplastic syndrome (MDS) and/or acute leukemia. The increasing identification of affected individuals and families has raised questions around the efficacy, timing, and optimal methods of surveillance. As part of the AACR Childhood Cancer Predisposition Workshop, an expert panel met to review the spectrum of leukemia-predisposing conditions, with the aim to develop consensus recommendations for surveillance for pediatric patients. The panel recognized that for several conditions, routine monitoring with complete blood counts and bone marrow evaluations is essential to identify disease evolution and enable early intervention with allogeneic hematopoietic stem cell transplantation. However, for others, less intensive surveillance may be considered. Because few reports describing the efficacy of surveillance exist, the recommendations derived by this panel are based on opinion, and local experience and will need to be revised over time. The development of registries and clinical trials is urgently needed to enhance understanding of the natural history of the leukemia-predisposing conditions, such that these surveillance recommendations can be optimized to further enhance long-term outcomes. (C) 2017 AACR.
dc.identifier.citationClinical Cancer Research, 2017; 23(11):14-22
dc.identifier.doi10.1158/1078-0432.CCR-17-0428
dc.identifier.issn1078-0432
dc.identifier.issn1557-3265
dc.identifier.orcidScott, H.S. [0000-0002-5813-631X]
dc.identifier.urihttps://hdl.handle.net/11541.2/127912
dc.language.isoen
dc.publisherAmerican Association for Cancer Research
dc.rightsCopyright 2017 American Association for Cancer Research Access Condition Notes: Preprint available on open access
dc.source.urihttps://doi.org/10.1158/1078-0432.ccr-17-0428
dc.subjectchildhood leukemia
dc.subjectgenetic predisposition
dc.subjectsurveillance
dc.subjectmyeldysplastic syndrome
dc.subjectbone marrow failure
dc.subjectHumans
dc.subjectLi-Fraumeni Syndrome
dc.subjectMyelodysplastic Syndromes
dc.subjectGenetic Predisposition to Disease
dc.subjectHematopoietic Stem Cell Transplantation
dc.subjectMutation
dc.subjectChild
dc.subjectTumor Suppressor Protein p53
dc.subjectLeukemia, Myeloid, Acute
dc.titleRecommendations for surveillance for children with leukemia-predisposing conditions
dc.typeJournal article
pubs.publication-statusPublished
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