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Myeloablative BU, fludarabine, antithymocyte globulin and low-dose TBI in the treatment of juvenile myelomonocytic leukaemia with allogeneic haematopoietic cell transplantation

Bone Marrow Transplantation volume 50, pages 455–456 (2015)Cite this article

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Juvenile myelomonocytic leukaemia (JMML) is a rare, aggressive myeloproliferative neoplasm that responds poorly to conventional chemotherapy.1, 2, 3 With the exception of rare cases of spontaneous remission seen in children with specific molecular mutations, allogeneic haematopoietic SCT (HSCT) is the only curative therapy. Myeloablative conditioning is widely considered to be essential to eradicate leukaemic clones and to faciliate engraftment. Owing to historically high rates of treatment-related mortality (TRM) in children with JMML, efforts are ongoing to develop newer approaches to HSCT therapy to reduce toxicities, including reduced-toxicity myeloablative conditioning regimens. We present pilot data on three children who underwent HSCT with such an approach.

A search of our institutional database was performed, and three patients were identified with a diagnosis of JMML who received a reduced-toxicity regimen. A retrospective chart review of the clinical records of the three cases was subsequently conducted to gather data. The database and clinical records were housed at Alberta Children’s Hospital in Calgary, Alberta, Canada. This retrospective study was approved by the University of Calgary Conjoint Health Research Ethics Board.

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Acknowledgements

We thank the patients and families, as well as the University of Calgary for funding Mr Moorjani’s summer studentship with our programme. We also thank Debbie O’Shea for her research assistant contributions.

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  1. University of Calgary, Section of Haematology/Oncology/Transplant, Alberta Children’s Hospital, Calgary, Canada

    G M T Guilcher, R Moorjani, T H Truong & V A Lewis

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  1. G M T Guilcher
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  2. R Moorjani
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  3. T H Truong
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  4. V A Lewis
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Correspondence to G M T Guilcher.

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Guilcher, G., Moorjani, R., Truong, T. et al. Myeloablative BU, fludarabine, antithymocyte globulin and low-dose TBI in the treatment of juvenile myelomonocytic leukaemia with allogeneic haematopoietic cell transplantation. Bone Marrow Transplant 50, 455–456 (2015). https://doi.org/10.1038/bmt.2014.272

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