Use of Rapamycin in a Patient With Juvenile Myelomonocytic Leukemia: A Case Report.

Shivani Y Upadhyay,Theodore B Moore,Satiro N De Oliveira

doi:10.1177/2324709617728528

Abstract

The relapse rate for children with juvenile myelomonocytic leukemia (JMML) status post hematopoietic stem cell transplantation (HSCT) approaches 50% within 5 years. Graft-versus-leukemia (GVL) is thought to play important role in the treatment of JMML. For this reason, careful management of immunosuppressive drugs after HSCT is crucial. This case report demonstrates that rapamycin and GVL represent a viable medical strategy for the management of pediatric patients with JMML who relapse following status post-HSCT.

Highlights

The standard treatment for Juvenile myelomonocytic leukemia (JMML) is chemotherapy followed by hematopoietic stem cell transplantation (HSCT), but the relapse rate for children with JMML status post-HSCT approaches 50% within 5 years[1,3,4] and remains the main cause of therapy failure and mortality
We describe an infant with JMML status postHSCT with evidence of graft loss and disease recurrence, but currently doing well following withdrawal of immunosuppression and initiation of rapamycin
The relapse rate for children with JMML status posttransplantation remains as high as 50% within 5 years.[1,3,17]

Summary

Introduction

Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative disorder that occurs in early childhood, often characterized by peripheral blood monocytosis, monosomy 7, dysplastic bone marrow, granulocyte-macrophage colonystimulating factor (GM-CSF) hypersensitivity, and increased fetal hemoglobin.[1,2] The standard treatment for JMML is chemotherapy followed by hematopoietic stem cell transplantation (HSCT), but the relapse rate for children with JMML status post-HSCT approaches 50% within 5 years[1,3,4] and remains the main cause of therapy failure and mortality.Progress has been made in understanding the molecular genetics and mechanisms of this disorder, implicating hyperactive RAS as essential initiating event.[5]. Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative disorder that occurs in early childhood, often characterized by peripheral blood monocytosis, monosomy 7, dysplastic bone marrow, granulocyte-macrophage colonystimulating factor (GM-CSF) hypersensitivity, and increased fetal hemoglobin.[1,2] The standard treatment for JMML is chemotherapy followed by hematopoietic stem cell transplantation (HSCT), but the relapse rate for children with JMML status post-HSCT approaches 50% within 5 years[1,3,4] and remains the main cause of therapy failure and mortality.

Results

Conclusion