Press Release | Frankfurt, 9 June 2023
Hematopoietic stem cell transplantation (HSCT) is a standard consolidation treatment for patients with relapsed or refractory acute lymphoblastic leukemia (R/R ALL). At present, it is difficult to predict the prognosis after HSCT in the R/R setting because of a paucity of available data. The HARMONY ALL research team has investigated data from 235 adult European patients with R/R ALL to shed light on the prognosis after HSCT. Alexander Russell-Smith (Pfizer) presents the results at the congress of the European Hematology Association.
Read the HARMONY #EHA2023 abstract >
Lack of data
Relapsed or refractory disease is an unfortunate reality for many adults with ALL. The disease can prove more challenging to treat in the R/R setting and is often associated with poorer outcomes. Undergoing HSCT is the goal for R/R ALL patients. However, it is difficult to predict survival and other outcomes after HSCT because the available datasets are scattered, frequently focus on de novo rather than R/R ALL, and often contain clinical information that is decades old. Using data from the unique HARMONY Big Data Platform, a research team has investigated data from 235 adult European patients with R/R ALL to obtain insight in the disease course after HSCT. Overall survival was assessed post-HSCT using Kaplan-Meier time-to-event analyses.
Russell-Smith: “The creation of the HARMONY Alliance has opened up a unique opportunity to answer a research question that has been outstanding for decades by gathering large amounts of data into one repository.”
Survival after transplant
The researchers observed that the one-year overall survival rate in the investigated patients was 45% and the five-year rate was 27%, from the point of transplant. Interestingly, the probability of survival after HSCT increased in time, with the probability of being alive one year after transplant in 2010-2019 (53%) being more than twice as high as in 1995-1999 (25%). Factors such as younger age at diagnosis and transplantation, and achieving negative minimal residual disease may positively influence post-HSCT survival. The researchers concluded that the data set has provided evidence for assumptions around what influences survival, but that post-HSCT outcomes remain unsatisfactory in adults with R/R ALL.
Russell-Smith: “Estimating medium term survival for these patients, and what factors influence that, is a great result from this research; it has made this project a success.”
Watch the interview at EHA2023 realized by VJHemOnc >
Rare disease in adults
ALL is a life-threatening disease if not treated immediately. The disease has a rapid onset and easily spreads to other organs. The treatment of ALL has dramatically improved in the past few decades, largely due to improvements in protocol design, supportive care, and risk stratification. The five-year event-free survival currently is more than 80% for children and 30-70% adults largely depending on age. ALL treatment protocols are intensive, typically involving multiple chemotherapeutic drugs administered over several months or years. Stem cell transplantation is often reserved for fit high-risk patients. In contrast to the other hematologic malignancies that are studied by the HARMONY Alliance, ALL occurs more frequently in children than adults.
HARMONY Big Data Platform
The HARMONY Alliance is a community with over 500 professionals, more than 120 partners/member organizations from 19 European countries, and a patient cluster with 9 European patient organizations. When HARMONY started in 2017, the hematology community was embarking on a journey towards embracing data sharing and recognizing the transformative power of big data. HARMONY set a crucial milestone when this endeavor was only starting. By laying the foundation for future collaborations, advancements, and discoveries that would incorporate state-of-the-art technologies in the field of hematology, HARMONY is making its contribution to improve the understanding and treatment of blood disorders.
Now, just six years later, we have transformed both the sharing and use of data. We have developed the Big Data Platform with a data lake that already contains data from 93,000 patients and has identified an additional 60,000 patient records that will be included in the months to come. All data is protected using a unique anonymization and security process. In more than 30 research projects, groundbreaking research is being carried out with the help of advanced customized data analytic services, including AI modelling.
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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).
HARMONY Coordination Office
Institute of Biomedical Research of
Salamanca (IBSAL)
Salamanca, Spain
HARMONY Communications
European Hematology Association (EHA)
The Hague, The Netherlands
