Conflict of interest: Nothing to declare.
Control of thrombotic thrombocytopenic purpura by sirolimus in a child with juvenile myelomonocytic leukemia and somatic N-RAS mutation
Article first published online: 3 MAR 2014
© 2014 Wiley Periodicals, Inc.
Pediatric Blood & Cancer
Volume 61, Issue 10, pages 1871–1873, October 2014
How to Cite
Maschan, M., Bobrynina, V., Khachatryan, L., Kalinina, I., Solopova, G., Avdonin, P., Nasedkina, T., Novichkova, G. and Maschan, A. (2014), Control of thrombotic thrombocytopenic purpura by sirolimus in a child with juvenile myelomonocytic leukemia and somatic N-RAS mutation. Pediatr. Blood Cancer, 61: 1871–1873. doi: 10.1002/pbc.25013
M.M. was the principal investigator and takes primary responsibility for the paper. V.B., P.A., T.N. and E.O. developed and implemented laboratory tests, acquired and interpreted laboratory results, L.K., I.K., G.S. cared for the patient at different time periods, acquired and interpreted clinical data, M.M., G.A. and A.M. drafted and revised the paper.
- Issue published online: 19 AUG 2014
- Article first published online: 3 MAR 2014
- Manuscript Accepted: 7 FEB 2014
- Manuscript Received: 18 JAN 2014
- autoimmune lymphoproliferative syndrome;
- juvenile myelomonocytic leukemia;
- thrombotic thrombopenic purpura
We describe an infant who developed juvenile myelomonocytic leukemia (JMML) at the age of 6 months. Myeloproliferation was effectively controlled by low-dose cytosine arabinoside and 13-cis retinoic acid therapy. Two years after therapy for JMML was stopped, at the age of 5 years, the patient developed autoimmune thrombotic thrombocytopenic purpura (TTP). TTP was transiently controlled by plasma exchange, prednisolone, rituximab, and cyclophosphamide, but relapsed within a short time. Long-term control of TTP was established by sirolimus. Somatic N-RAS G38AGly13Asp substitution was restricted to hematopoietic cells. The somatic N-RAS mutation may link myeloproliferation and autoimmunity. Pediatr Blood Cancer 2014; 61:1871–1873. © 2014 Wiley Periodicals, Inc.