Azacitidine and Sorafenib Therapy in a Pediatric Patient With Refractory Acute Myeloid Leukemia With Monosomy 7 and Somatic PTPN11 Mutation

Nathan A Dahl; Samantha T Michaels; Richard L McMasters; Sharat Chandra; Maureen M O'Brien

doi:10.1002/pbc.25805

Azacitidine and Sorafenib Therapy in a Pediatric Patient With Refractory Acute Myeloid Leukemia With Monosomy 7 and Somatic PTPN11 Mutation

Pediatr Blood Cancer. 2016 Mar;63(3):551-3. doi: 10.1002/pbc.25805. Epub 2015 Oct 20.

Authors

Nathan A Dahl¹, Samantha T Michaels², Richard L McMasters³, Sharat Chandra², Maureen M O'Brien²

Affiliations

¹ Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio.
² Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio.
³ Division of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio.

PMID: 26485542
DOI: 10.1002/pbc.25805

Abstract

Monosomy 7 is a well-documented cytogenetic aberration in pediatric acute myeloid leukemia (AML) and may occur in combinations with molecular abnormalities including PTPN11 mutation. PTPN11 mutations contribute to leukemogenesis through upregulation of Ras pathway signaling. We present the case of a 3-year-old female with AML with monosomy 7 and somatic PTPN11 mutation who was refractory to conventional AML chemotherapy but responded to a novel regimen of azacitidine and sorafenib followed by stem cell transplantation. Combination therapy with azacitidine and sorafenib may be an effective therapeutic strategy for patients with AML with Ras pathway abnormalities.

Keywords: AML; new agents; pediatric oncology.

Publication types

Case Reports

MeSH terms

Antimetabolites, Antineoplastic / administration & dosage*
Antineoplastic Agents / administration & dosage*
Azacitidine / administration & dosage*
Child, Preschool
Chromosome Deletion*
Chromosomes, Human, Pair 7
Drug Therapy, Combination
Female
Humans
Leukemia, Myeloid, Acute / drug therapy*
Leukemia, Myeloid, Acute / genetics*
Mutation
Niacinamide / administration & dosage
Niacinamide / analogs & derivatives*
Phenylurea Compounds / administration & dosage*
Protein Tyrosine Phosphatase, Non-Receptor Type 11 / genetics*
Sorafenib
Stem Cell Transplantation

Substances

Antimetabolites, Antineoplastic
Antineoplastic Agents
Phenylurea Compounds
Niacinamide
Sorafenib
PTPN11 protein, human
Protein Tyrosine Phosphatase, Non-Receptor Type 11
Azacitidine

Supplementary concepts

Chromosome 7, monosomy