Subclonal mutations in SETBP1 confer a poor prognosis in juvenile myelomonocytic leukemia

Author:

Stieglitz Elliot1ORCID,Troup Camille B.2,Gelston Laura C.1,Haliburton John3,Chow Eric D.4,Yu Kristie B.1,Akutagawa Jon1,Taylor-Weiner Amaro N.5,Liu Y. Lucy6,Wang Yong-Dong7,Beckman Kyle1,Emanuel Peter D.6,Braun Benjamin S.1,Abate Adam3,Gerbing Robert B.8,Alonzo Todd A.9ORCID,Loh Mignon L.110

Affiliation:

1. Department of Pediatrics, Benioff Children’s Hospital, University of California, San Francisco, San Francisco, CA;

2. Bio-Rad Laboratories, Hercules, CA;

3. Department of Bioengineering and Therapeutic Sciences, California Institute for Quantitative Biosciences, University of California, San Francisco, San Francisco, CA;

4. Center for Advanced Technology, Department of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA;

5. Broad Institute, Cambridge, MA;

6. Winthrop P. Rockefeller Cancer Institute, University of Arkansas for Medical Sciences, Little Rock, AR;

7. Department of Computational Biology, St. Jude Children’s Research Hospital, Memphis, TN;

8. Children's Oncology Group, Monrovia, CA;

9. Keck School of Medicine, University of Southern California, Los Angeles, CA; and

10. Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, CA

Abstract

Key Points Mutations in SETBP1 can be detected using droplet digital polymerase chain reaction in at least 30% of patients with JMML and are associated with a dismal prognosis. Patients harboring rare cells with mutant SETBP1 at diagnosis should be considered candidates for swift hematopoietic stem cell transplant.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Reference38 articles.

1. Recent advances in the pathogenesis and treatment of juvenile myelomonocytic leukaemia.;Loh;Br J Haematol,2011

2. Juvenile myelomonocytic leukemia: molecular pathogenesis informs current approaches to therapy and hematopoietic cell transplantation.;Dvorak;Front Pediatr,2014

3. Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial.;Locatelli;Blood,2005

4. Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: a report from the Children’s Oncology Group [published online ahead of print December 8, 2014].;Stieglitz;Pediatr Blood Cancer

5. Loss of the normal NF1 allele from the bone marrow of children with type 1 neurofibromatosis and malignant myeloid disorders.;Shannon;N Engl J Med,1994

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