title: How have advances in our understanding of the molecular genetics of paediatric leukaemia led to improved targeted therapies for these diseases?
abstract: The term "leukaemia" refers to a large and heterogenous group of diseases, with treatment response and outcome dependent on the specific type of malignancy. New molecular methods allow us to specifically evaluate the type of disorder, and provide treatment of necessary intensity. The aim of this review is to provide insight into the progress in leukaemia treatment that had been possible due to advances in molecular genetics over the last few decades. Those new sophisticated diagnostic methods have allowed us not only to predict patients' prognosis but also to provide a specific therapy depending on the molecular and genetic characteristics of patients. Our review is based on 25 articles regarding novel diagnostic and therapeutic methods as well as prognostic factors, released between 1992 and 2011. Those articles focus mostly on molecular and cytogenetic testing allowing revolutionary methods of patient classification and individual therapy for this highly heterogeneous group of disorders. Implementation of molecular genetic testing to evaluate the type of leukaemia allowed paediatric oncologists and haematologists to adjust the intensity of treatment, improve outcome, minimize toxicity of therapies and considerably lower the risk of side effects. In the last few decades there has been a great improvement in survival among children suffering from haematopoietic malignancies. Progress made in molecular genetics allowed the creation of new treatment protocols that are designed to maintain a high cure rate for children with leukaemia while reducing toxicity.
authors: Haematology and Oncology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors: Elwira
authors: E
authors: Szychot
authors: Department of Pediatrics, Nephrology with Dializotheraphy and Management of Acute Poisoning, Pomeranian Medical University, Szczecin, Poland.
authors: Andrzej
authors: A
authors: Brodkiewicz
authors: Department of Pediatrics, Hematology and Pediatric Oncology, Pomeranian Medical University, Szczecin, Poland.
authors: Jarosław
authors: J
authors: Peregud-Pogorzelski
mesh_terms: D000818:Animals; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D014408:Biomarkers, Tumor / Q000235:genetics*; D002648:Child; D020022:Genetic Predisposition to Disease; D006801:Humans; D007938:Leukemia / Q000188:drug therapy* / Q000235:genetics* / Q000378:metabolism; D025202:Molecular Diagnostic Techniques; D058990:Molecular Targeted Therapy / Q000009:adverse effects; D010641:Phenotype; D057285:Precision Medicine; D011237:Predictive Value of Tests; D012074:Remission Induction; D015398:Signal Transduction / Q000187:drug effects*; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D014408:Biomarkers, Tumor