Low dose decitabine in very high risk relapsed or refractory acute myeloid leukaemia in children and young adults
Christine L Phillips,Stella M. Davies,Richard McMasters,Michael J. Absalon,Maureen M. O'Brien,Jun Mo,Randall Broun,Jeffrey A. Moscow,Teresa A. Smolarek,Ramiro Garzon,William Blum,Sebastian Schwind,Guido Marcucci,John P. Perentesis +13 more
TLDR
Low‐dose decitabine has encouraging activity and tolerability in adults with acute myeloid leukaemia (AML), but paediatric experience is lacking, and eight patients with refractory/relapsed AML are reported on.Abstract:
Low-dose decitabine has encouraging activity and tolerability in adults with acute myeloid leukaemia (AML), but paediatric experience is lacking. We report our retrospective experience with decitabine in eight children and young adults (median age 4 years) with refractory/relapsed AML, who had failed multiple regimens or were not candidates for standard retrieval regimens due to prior toxicities. Three of eight patients (38%) had complete response (CR; 1 each of CR, CR with incomplete platelet recovery and CR with incomplete count recovery). Best responses were observed after a median of 2.5 cycles (range 1-4 cycles). Four patients received subsequent allogeneic stem cell transplant, and two remain in long-term CR.read more
Citations
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Pediatric AML: From Biology to Clinical Management
TL;DR: These efforts towards future personalized therapy in a rare disease, such as pediatric AML, require intensive international collaboration in order to enhance the survival rates of Pediatrics, while aiming to reduce long-term toxicity.
Journal ArticleDOI
Genetic predisposition to myelodysplastic syndrome and acute myeloid leukemia in children and young adults
TL;DR: A practical algorithm for approaching a patient with a suspected MDS/AML predisposition, and an in-depth review of the established and emerging familial MDS-AML syndromes caused by mutations in the ANKRD26, CEBPA, DDX41, ETV6, GATA2, RUNX1, SRP72 genes are provided.
Journal ArticleDOI
Oncogenic Roles and Inhibitors of DNMT1, DNMT3A, and DNMT3B in Acute Myeloid Leukaemia.
TL;DR: Hypermethylation of tumour suppressors mediated by DNMT1 or DNMT3B contributes to the progression and severity of AML (except MLL-AF9 and inv(16)(p13;q22) AML for DN MT3B), while mutation affecting DNMT2A represents an early genetic lesion in the pathogenesis of AMl.
Journal ArticleDOI
5-Azacytidine and DLI can induce long-term remissions in AML patients relapsed after allograft
J Steinmann,Hartmut Bertz,Ralph Wäsch,Reinhard Marks,Robert Zeiser,Lioudmila Bogatyreva,Jürgen Finke,Michael Lübbert +7 more
TL;DR: Long-term remissions can be induced by this well-tolerated outpatient treatment, particularly in patients without peripheral blood blasts, as well as upregulating Ags on blasts before DLI.
Journal ArticleDOI
Treatment of relapsed AML and MDS after allogeneic stem cell transplantation with decitabine and DLI—a retrospective multicenter analysis on behalf of the German Cooperative Transplant Study Group
Thomas Schroeder,Christina Rautenberg,William Krüger,Uwe Platzbecker,Gesine Bug,Juliane Steinmann,Stefan Klein,Olaf Hopfer,Kathrin Nachtkamp,Mustafa Kondakci,Stefanie Geyh,Rainer Haas,Ulrich Germing,Martin Bornhäuser,Guido Kobbe +14 more
TL;DR: DAC exerts clinical efficacy in patients with AML or MDS relapsing after allo-SCT and is able to induce durable remissions in individual patients suggesting that DAC may be an alternative to Aza or even a second choice after Aza failure.
References
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Ramiro Garzon,Shujun Liu,Muller Fabbri,Zhongfa Liu,Catherine E. A. Heaphy,Elisa Callegari,Sebastian Schwind,Jiuxia Pang,Jianhua Yu,Natarajan Muthusamy,Violaine Havelange,Stefano Volinia,William Blum,Laura J. Rush,Danilo Perrotti,Michael Andreeff,Clara D. Bloomfield,John C. Byrd,Kenneth Chan,Lai-Chu Wu,Carlo M. Croce,Guido Marcucci +21 more
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Results of a randomized study of 3 schedules of low-dose decitabine in higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia
Hagop M. Kantarjian,Yasuhiro Oki,Guillermo Garcia-Manero,Xuelin Huang,Susan O'Brien,Jorge E. Cortes,Stefan Faderl,Carlos E. Bueso-Ramos,Farhad Ravandi,Zeev Estrov,Alessandra Ferrajoli,William G. Wierda,Jianqin Shan,Jan Davis,Francis J. Giles,Hussain I. Saba,Jean Pierre J. Issa +16 more
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