Abstract
Background: Juvenile Myelomonocytic leukemia(JMML) is a rare
and aggressive malignancy found in children. The genomic landscape of
the JMML shows that the most common mutated genes found in the RAS. The
risk stratification and the management of JMML patients is determined by
the precise evaluation of the underlying genetic mutations. The
co-occurring mutations along with the RAS pathway mutations may affect
the outcomes of the disease. PTPN11 is the most common mutation
found in JMML. In this study, we describe the outcomes of JMML patients
who had an underlying PTPN11 mutation along with a mutation in
the SETBP1 gene. Methods: DNA was extracted from the 43
cases with JMML after confirmation of the diagnosis. Whole exome
sequencing was performed to find out the underlying germline and somatic
mutations. Results: We found that about 35%(n=14) of patients
harboured a PTPN11 somatic mutation. A coexisting SETBP1
mutation was found in 5 patients out of 14 cases. In our cohort of
patients, we found that the SETBP1 was exclusively associated
with PTPN11 and all 5 patients transformed into AML. The median
time to AML transformation was 12 months (13 days-35 months).
PTPN11 mutation with co-existing SETBP1 mutation showed a
worse outcome compared to other PTPN11 positive patients and all 5
patients died within 3 months of transformation. Conclusion: In
with PTPN11 positive JMML a coexisting SETBP1 mutation confers a
poorer prognosis. These patients have a high risk of AML transformation.
These patients should be candidate for consideration of early
hematopoietic stem cell transplantation (HSCT).