MDS-3: Differential outcomes of patients with MDS undergoing HSCT vs non-transplant strategies

Challenge

Allogeneic hematopoietic stem cell transplant (HSCT) is the only treatment available for myelodysplastic neoplasms/syndromes (MDS), a disease that primarily affects older people (>70 years). Although newer transplant strategies have allowed older patients to benefit from HSCT, only 15% of MDS patients receive this treatment. This is due to a lack of consensus about which patients benefit most from HSCT as well as the exact timing of the treatment. Finding consensus is difficult because MDS is a complex and highly heterogeneous spectrum of disorders, and it is not clear which subcategories respond best, if at all, to HSCT.

Project Partnership

EBMT

Project Leadership

Carmelo Gurnari, Tor Vergata, University of Rome, Italy
Marie Robin, Hôpital Saint Louis Paris, France
Donal McLornan, University College London Hospital, London, UK

Impact

Providing specific data on outcomes of MDS patients who undergo transplant vs non-transplant approaches could help clarify which patients benefit most from transplant.
Filling in knowledge gaps about the best use of general MDS risk scores and transplant interactions could facilitate a better selection of patients for HSCT.

Project Summary

Older (aged over 70 years) MDS patients could benefit from HCST. However, this treatment option is underutilized due to a lack of consensus about which patients benefit from this treatment as well as the treatment’s exact timing. Therefore, this study aims to provide specific data on MDS patient outcomes for transplant versus nontransplant approaches and fill in knowledge gaps concerning the best use of general MDS risk scores and transplant interactions.

HARMONY and EBMT datasets will be leveraged for this study. The HSCT cohort will be all adult patients (aged >50 years) in the EBMT registry who received a bone marrow or peripheral blood allogeneic stem cell transplantation for MDS (excluding CMML) in the period 2011-2021. The non-transplant cohort will be MDS patients enrolled in the HARMONY initiative. Bias inherent to studies on patient populations whose characteristics may determine the different treatment approaches will be avoided by adopting a paired-matched strategy to analyze patients with similar age, gender, performance status, and IPSS-R/M risk categories. Baseline recipient data, transplant characteristics, baseline donor data, and follow-up recipient data will be analyzed.

The overall objectives are:

Determine if and to what extent HSCT benefits different subcategories of MDS patients compared to non-transplant strategies.
Determine if HSCT pre-treatment benefits higher-risk (IPSS-R and IPSS-M) MDS cases.

The paired match analysis of a large cohort of treated and non-treated MDS patient outcomes should provide clearer insights into the potential benefit of HSCT for different subcategories of MDS patients. These insights, combined with a better understanding of how to use general MDS risk scores and the possible benefits of HSCT pre-treatment, will hopefully contribute to a greater consensus about the use of HSCT to treat MDS.

Public-private partnership for Big Data in Hematology

HARMONY and HARMONY PLUS are funded through the Innovative Medicines Initiative (IMI), Europe's largest public-private initiative aiming to speed up the development of better and safer medicines for patients. Funding is received from the IMI 2 Joint Undertaking and is listed under grant agreement for HARMONY No. 116026 and grant agreement for HARMONY PLUS No. 945406. This Joint Undertaking receives support from the European Union’s Horizon 2020 Research and Innovation Programme and the European Federation of Pharmaceutical Industries and Associations (EFPIA).