By Emanuele Angelucci, HWP Chair
This year’s Annual Meeting in Florence marked a significant milestone for the Haemoglobinopathies Working Party (HWP), which has now been firmly established as an integral part of the EBMT. The educational and scientific sessions were of outstanding quality and drew large audiences, filling some of the venue’s largest halls. Interest was also clearly evident during the business meetings.
This momentum is likely to continue growing in the coming years, particularly as gene therapy becomes more accessible and efforts to reach stakeholders in resource-limited settings begin to yield results. These accomplishments are a testament to our collaborative efforts, and we should all take pride in them.
At the heart of the society's work—and essential to sustaining the activities of the Working Party—is scientific output. This, in turn, depends on the quality and completeness of the data within the EBMT Registry. Unfortunately, the current status of data entry for haemoglobinopathies is suboptimal and does not allow the completion of all six approved studies, including one supported by an external grant. While we are actively evaluating new scientific proposals and entering discussions with external partners, our ability to move forward is contingent upon improved data completeness.
Despite repeated and sustained efforts, progress has been limited due to the heavy burden placed on a small number of centres. This underscores the vital role of the HWP as a custodian for these disorders and highlights the urgent, one-time need to address this legacy challenge if we are to ensure future success.
In light of this, the HWP has obtained approval from the EBMT Executive Committee to allocate financial resources accrued through its activities toward a data completion initiative. This will be a one-off effort specifically for the HWP and will not be repeated in the future. Due to limited resources—both financial and human—as well as varying regulatory requirements across countries, the initiative will only be targeted at those centres from which the most complete and impactful data can be obtained, within a tight timeframe.
Following careful analysis, and for economic reasons, the HWP Board has approved focusing on the largest centres in most of the key countries, which together account for approximately 70% of the HWP Registry activity.
Funding will be provided for fully completed questionnaires only and will be allocated as follows:
● €50 per patient for the reduced questionnaire (306 variables), the minimum dataset required for study eligibility.
● €100 per patient for the extended questionnaire (353 variables, including chimerism data).
Centres may choose the questionnaire they are able to complete, based on their capacity and data availability.
During implementation, we will remain fully aligned with the EBMT’s obligations as a Dutch registered non-profit organisation and ensure compliance with EU and international financial regulations.
In the coming days, we will contact the selected centres directly with detailed instructions, timelines, and data submission procedures. A dedicated point of contact will also be provided to assist with any questions or logistical issues. We encourage all participating centres to begin reviewing their existing data and preparing for this effort, as the timeline will be limited and strictly enforced.
Thank you for your continued support and commitment to advancing the field.