Research Project: Improve treatment of patients with Acute Promyelocytic Leukemia

This study will assess how doctors can best manage patients with Acute Promyelocytic Leukemia (APL), particularly during the early stages of treatment.

APL is a very rare form of leukemia and, although treatment has advanced in recent years, important questions have yet to answered. This is because the small number of patients included in clinical studies makes it difficult to work out the best strategies for managing the disease. The HARMONY Data Platform is a powerful database that will allow us to combine and analyze information on a large number of APL patients - thereby shedding light on some of these key unanswered questions.

Answering the key questions in APL: APL is an aggressive form of leukemia, but it is also very rare - affecting only a handful patients. APL used to be fatal in most cases, but treatment has advanced enormously over the last decade thanks to intensive research into the disease. As a result, around 9 out of every 10 APL patients can now be cured using targeted treatments alone or in combination with chemotherapy.

Although the treatment of APL has been revolutionized by these use new targeted drugs, important unanswered questions about the clinical care of the disease remain. In particular, doctors are still not sure about the best way to prevent and manage complications.

The HARMONY Big Data Platform will bring together data on APL patients from clinical studies carried out across Europe. This will allow large amounts of information to be analyzed quickly and effectively. The study will compare data on patients receiving different treatments to try and work out which therapy gives the best outcomes. A particular focus will be on how best to prevent and treat complications that can occur in APL patients. These complications include blood disorders, disease relapses that affect the central nervous system and severe side effects of treatment.

Other key questions in APL that the HARMONY Big Data Platform will try to answer include:

The role and potential survival advantage of maintenance therapy - where patients receive ongoing treatment to try and stop their disease coming back

The ideal choice of therapy for elderly patients.

Looking to the future, the new knowledge gained from this study will help to improve the everyday management of APL, ensuring patients get the best possible outcomes.

What will be the impact?

By addressing some of the key issues in APL that are not fully understood, this study will help to optimize the everyday clinical care of patients. It may also lead to updates and improvements to current treatment guidelines - particularly when it comes to the best strategies for preventing and treating complications.

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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).