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EBMT 2021 | Novel prognostic score for CMML patients undergoing SCT

Nico Gagelmann, MD, University Medical Center Hamburg-Eppendorf, Hamburg, Germany, discusses the development of a new prognostic score, which includes mutation profile and clinical features, for patients with chronic myelomonocytic leukemia (CMML) who are undergoing stem cell transplantation (SCT). 240 patients were included in the study with a median follow-up of 5.5 years. Mutations in ASXL1 and/or NRAS were found to be significantly associated with worse outcomes. This interview took place during the 47th Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT) 2021.

Transcript (edited for clarity)

Our rationale for this study on a prognostic score for CMML patients, undergoing stem cell transplantation was basically the main risk stratifications, for these patients exist for the non-transplant setting. So, for patients who are newly diagnosed. For these patients, we have several risk stratification systems, the CPSS, or the molecular enhanced CPSS, the CPSS-Mol, there is the Mayo model and the French model from the GFM community...

Our rationale for this study on a prognostic score for CMML patients, undergoing stem cell transplantation was basically the main risk stratifications, for these patients exist for the non-transplant setting. So, for patients who are newly diagnosed. For these patients, we have several risk stratification systems, the CPSS, or the molecular enhanced CPSS, the CPSS-Mol, there is the Mayo model and the French model from the GFM community. But patients who get transplanted were found to have a difficult risk stratification over the last 10 years.

So there were EBMT studies, and CIBMTR studies, which identified no significant impact of cytogenetics for instance, or for specific patient profiles with clinical factors. And then what we wanted to address was the combined effect of clinical and molecular genetic information. And that’s why we gathered a huge cohort of 240 patients with clinical and molecular information. This is the largest CMML patient cohort in transplant setting with this information to date. And we collaborated with several centers from Germany, from the Netherlands, from France, and even from the US to do this study, and we are very, very grateful for this collaboration.

And we then found basically three risk factors. One was the comorbidity index at time of transplantation, the blast percentage at time of transplantation, and ASXL1, and or NRAS mutated genotype. And these three factors could build a seminal transplant score, ranging up to 20 points. And then we could stratify at a maximum of five risk groups with highest risk and lowest risk.

And that was basically the whole rationale, to identify patients who could benefit from transplant, but even patients might not benefit for transplant, and who might be interesting subpopulations for future research in the non-transplant field. That was basically our study. We created a website, a tool which could be used freely for everyone to use, to facilitate patient counseling. And we look forward to external validation and to continuous use, and feedback from the clinics to even further refine, or just validate the score.

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