title: Treatment of acute leukemia in children with ataxia telangiectasia (A-T).
abstract: Early onset ataxia telangiectasia (A-T) is a neurodegenerative DNA-instability disorder, which presents early in childhood. Hallmarks of A-T are progressive ataxia and a dramatic increased risk of developing malignancies (25%), especially of hematological origin. In children these malignancies mainly concern aggressive Non-Hodgkin lymphoma, acute leukemias and Hodgkin lymphoma. Of the acute leukemias, T-cell lymphoblastic leukemia (T-ALL) is by far the most common. Since patients with A-T experience increased toxicity to radio- and chemotherapeutic treatment, the optimal treatment strategy of acute leukemia remains subject of debate. Review of literature of treatment of T-ALL in patients with A-T (n = 18) showed that many patients are not diagnosed with A-T at time of presentation of T-ALL. This implicates that physicians must be aware of symptoms of A-T in young patients presenting with T-ALL. Complete remission rates are high following upfront modified as well as unmodified treatment strategies. Treatment of ALL in children with A-T is feasible and should be performed. Definitive treatment strategy must be determined by shared decision making with patient, caretakers and medical team. Future prospective studies are needed to elucidate optimal treatment strategy.
authors.affiliation: Department of Pediatrics, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.
authors.forename: M H D
authors.initials: MH
authors.lastname: Schoenaker
authors.affiliation: Department of Hematology and French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Laboratory of Molecular Mechanisms of Hematologic Disorders and Therapeutic Implication, INSERM UMR 1163 & CNRS ERL 8254, France; Institut Imagine, Sorbonne Paris Cité, Paris Descartes University, France.
authors.forename: F
authors.initials: F
authors.lastname: Suarez
authors.affiliation: Department of Pediatric Hematology and Oncology, Zabrze, Medical University of Silesia, Katowice, Poland.
authors.forename: T
authors.initials: T
authors.lastname: Szczepanski
authors.affiliation: Pediatric Immuno-Haematology and Rheumatology Unit and French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, INSERM U1163, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Paris Descartes University, Sorbonne Paris Cité, Imagine Institute, Paris, France.
authors.forename: N
authors.initials: N
authors.lastname: Mahlaoui
authors.affiliation: Department of Pediatric Oncology and Hematology, Sophia Children's Hospital, Erasmus MC, Postbox 2040, 3000CA Rotterdam, Netherlands. Electronic address: j.loeffen@erasmusmc.nl.
authors.forename: J L
authors.initials: JL
authors.lastname: Loeffen
mesh_terms: D001260:Ataxia Telangiectasia / Q000150:complications / Q000188:drug therapy* / Q000453:epidemiology* / Q000473:pathology; D002648:Child; D018572:Disease-Free Survival; D006801:Humans; D054218:Precursor T-Cell Lymphoblastic Leukemia-Lymphoma / Q000150:complications / Q000188:drug therapy* / Q000453:epidemiology* / Q000473:pathology; D011446:Prospective Studies
keywords: Ataxia telangiectasia; Cancer; Children; Leukemia; Malignancy