Literature DB >> 33369023

Venetoclax/decitabine for a pediatric patient with chronic myelomonocytic leukemia.

John C Molina1,2, Julie M Asare2, Laura Tuschong3, Robert R West3, Katherine R Calvo4, Rebecca Persky5,6, Alison M Boyce7, Dima A Hammoud8, Steven M Holland9, Dennis Hickstein3, Nirali N Shah1.   

Abstract

Chronic myelomonocytic leukemia (CMML) is a myelodysplastic syndrome (MDS)/myeloproliferative disorder most commonly seen in the elderly. We describe an adolescent with monosomy 7 CMML presenting as central diabetes insipidus (DI), who was treated with venetoclax and decitabine as a bridge to hematopoietic stem cell transplantation (HSCT). Central DI is a rare manifestation of monosomy 7-associated MDS including CMML, itself a rare manifestation of GATA2 deficiency, particularly in children. Venetoclax/decitabine was effective for treatment of CMML as a bridge to HSCT.
© 2020 Wiley Periodicals LLC.

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Year:  2020        PMID： 33369023      PMCID： PMC9357463          DOI： 10.1002/pbc.28865

Source DB:  PubMed          Journal:  Pediatr Blood Cancer        ISSN： 1545-5009            Impact factor:   3.838


  31 in total

Review 1.  GATA2 deficiency.

Authors:  Amy P Hsu; Lisa J McReynolds; Steven M Holland
Journal:  Curr Opin Allergy Clin Immunol       Date:  2015-02

Review 2.  Peripheral blood stem cell versus bone marrow transplantation: A perspective from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.

Authors:  Michael Byrne; Bipin N Savani; Mohamad Mohty; Arnon Nagler
Journal:  Exp Hematol       Date:  2016-04-19       Impact factor: 3.084

3.  A phase II study of the efficacy, safety, and determinants of response to 5-azacitidine (Vidaza®) in patients with chronic myelomonocytic leukemia.

Authors:  Srinivas K Tantravahi; Philippe Szankasi; Jamshid S Khorashad; Kim-Hien Dao; Tibor Kovacsovics; Todd W Kelley; Michael W Deininger
Journal:  Leuk Lymphoma       Date:  2016-02-08

4.  HSCT for GATA2 deficiency across the pond.

Authors:  Dennis Hickstein
Journal:  Blood       Date:  2018-03-22       Impact factor: 22.113

Review 5.  Monosomy 7 predisposes to diabetes insipidus in leukaemia and myelodysplastic syndrome.

Authors:  A de la Chapelle; R Lahtinen
Journal:  Eur J Haematol       Date:  1987-11       Impact factor: 2.997

6.  Concomitant inhibition of DNA methyltransferase and BCL-2 protein function synergistically induce mitochondrial apoptosis in acute myelogenous leukemia cells.

Authors:  Twee Tsao; Yuexi Shi; Steven Kornblau; Hongbo Lu; Sergej Konoplev; Ansu Antony; Vivian Ruvolo; Yi Hua Qiu; Ninaxiang Zhang; Kevin R Coombes; Michael Andreeff; Kensuke Kojima; Marina Konopleva
Journal:  Ann Hematol       Date:  2012-08-15       Impact factor: 3.673

7.  Safety and preliminary efficacy of venetoclax with decitabine or azacitidine in elderly patients with previously untreated acute myeloid leukaemia: a non-randomised, open-label, phase 1b study.

Authors:  Courtney D DiNardo; Keith W Pratz; Anthony Letai; Brian A Jonas; Andrew H Wei; Michael Thirman; Martha Arellano; Mark G Frattini; Hagop Kantarjian; Relja Popovic; Brenda Chyla; Tu Xu; Martin Dunbar; Suresh K Agarwal; Rod Humerickhouse; Mack Mabry; Jalaja Potluri; Marina Konopleva; Daniel A Pollyea
Journal:  Lancet Oncol       Date:  2018-01-12       Impact factor: 41.316

8.  Treatment with Hypomethylating Agents before Allogeneic Stem Cell Transplant Improves Progression-Free Survival for Patients with Chronic Myelomonocytic Leukemia.

Authors:  Piyanuch Kongtim; Uday Popat; Antonio Jimenez; Sameh Gaballa; Riad El Fakih; Gabriela Rondon; Julianne Chen; Carlos Bueso-Ramos; Gautam Borthakur; Naveen Pemmaraju; Guillermo Garcia-Manero; Hagop Kantarjian; Amin Alousi; Chitra Hosing; Paolo Anderlini; Issa F Khouri; Partow Kebriaei; Borje S Andersson; Betul Oran; Katayoun Rezvani; David Marin; Elizabeth J Shpall; Richard E Champlin; Stefan O Ciurea
Journal:  Biol Blood Marrow Transplant       Date:  2015-09-04       Impact factor: 5.742

9.  GATA2 deficiency: a protean disorder of hematopoiesis, lymphatics, and immunity.

Authors:  Michael A Spinner; Lauren A Sanchez; Amy P Hsu; Pamela A Shaw; Christa S Zerbe; Katherine R Calvo; Diane C Arthur; Wenjuan Gu; Christine M Gould; Carmen C Brewer; Edward W Cowen; Alexandra F Freeman; Kenneth N Olivier; Gulbu Uzel; Adrian M Zelazny; Janine R Daub; Christine D Spalding; Reginald J Claypool; Neelam K Giri; Blanche P Alter; Emily M Mace; Jordan S Orange; Jennifer Cuellar-Rodriguez; Dennis D Hickstein; Steven M Holland
Journal:  Blood       Date:  2013-11-13       Impact factor: 22.113

10.  Central diabetes insipidus: an unusual complication in a child with juvenile myelomonocytic leukemia and monosomy 7.

Authors:  Pacharapan Surapolchai; Shau-Yin Ha; Godfrey Chi-Fung Chan; Johannes B Lukito; Thomas S K Wan; Chi-Chiu So; Alan Kwok-Shing Chiang
Journal:  J Pediatr Hematol Oncol       Date:  2013-03       Impact factor: 1.289
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  2 in total

1.  Donor source and post-transplantation cyclophosphamide influence outcome in allogeneic stem cell transplantation for GATA2 deficiency.

Authors:  Diana X Nichols-Vinueza; Mark Parta; Nirali N Shah; Jennifer M Cuellar-Rodriguez; Thomas R Bauer; Robert R West; Amy P Hsu; Katherine R Calvo; Seth M Steinberg; Luigi D Notarangelo; Steven M Holland; Dennis D Hickstein
Journal:  Br J Haematol       Date:  2021-09-27       Impact factor: 8.615

2.  ASXL1 and STAG2 are common mutations in GATA2 deficiency patients with bone marrow disease and myelodysplastic syndrome.

Authors:  Robert R West; Katherine R Calvo; Lisa J Embree; Weixin Wang; Laura M Tuschong; Thomas R Bauer; Desiree Tillo; Justin Lack; Stephenie Droll; Amy P Hsu; Steven M Holland; Dennis D Hickstein
Journal:  Blood Adv       Date:  2022-02-08
  2 in total

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