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

Upadhyay, Shivani; Oliveira, Satiro N. De; Moore, Theodore B.

doi:10.1177/2324709617728528

Cited by 2 publications

(1 citation statement)

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“…A case report documented on a 9‐year old patient with JMML experiencing a relapse 9 months after HSCT with development of GVHD, which was controlled with rapamycin. Interestingly, 77 months later, the patient was still alive and in remission, raising the possibility that besides the immunomodulating effect, rapamycin also excerted an antileukemic effect 87 …”

Section: Novel Targeted Appoaches In Jmmlmentioning

confidence: 99%

Targeted therapy in juvenile myelomonocytic leukemia: Where are we now?

Vos

Hofmans

Lammens

et al. 2022

Pediatric Blood & Cancer

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Juvenile myelomonocytic leukemia (JMML) is a rare and aggressive clonal neoplasm of early childhood, classified as an overlap myeloproliferative/myelodysplastic neoplasm by the World Health Organization. In 90% of the patients with JMML, typical initiating mutations in the canonical Ras pathway genes NF1, PTPN11, NRAS, KRAS, and CBL can be identified. Hematopoietic stem cell transplantation (HSCT) currently is the established standard of care in most patients, although long‐term survival is still only 50–60%. Given the limited therapeutic options and the important morbidity and mortality associated with HSCT, new therapeutic approaches are urgently needed. Hyperactivation of the Ras pathway as disease mechanism in JMML lends itself to the use of targeted therapy. Targeted therapy could play an important role in the future treatment of patients with JMML. This review presents a comprehensive overview of targeted therapies already developed and evaluated in vitro and in vivo in patients with JMML.

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Section: Novel Targeted Appoaches In Jmmlmentioning

confidence: 99%