mAPPHemo

mAPPHemo monitors adverse effects from the use of drugs for hemophilia treatment.

Hemophilia is a congenital coagulation disorder caused by the absence or insufficient production of clotting factor VIII (FVIII) in hemophilia A and of clotting factor IX (FIX) in hemophilia B. The symptoms are excessive or unexplained bleeding whose severity generally corresponds to the extent of the FVIII/FIX deficit. Hemophilia can range from severe (spontaneous bleeding, mostly into the muscles and joints, starting in early childhood) to moderate or mild, where bleeding mostly results from injury, surgery, or invasive procedures.

Hemophilia is treated by injecting a concentrate into the bloodstream to replace the deficient clotting factor. These products are either plasma-derived or recombinant, and can be used on demand (to resolve a bleeding episode in course) or prophylactically (at regular intervals to prevent bleeding episodes). In recent years, researchers have explored innovative solutions with a view to developing a new generation of treatment products. We are living in truly historical times for hemophilia treatment, and with an ever-growing number of new drugs coming into widespread use, we need to be extra vigilant that they are safe and effective.

mAPPHemo, developed to the specifications of the Factor VIII, Factor IX and Rare Coagulation Disorders Subcommittee of the International Society on Thrombosis and Haemostasis (ISTH), collects data on the use of these drugs and any side effects experienced by patients. It can be used by individual healthcare facilities or on a region- or nationwide level, allowing clinicians to gather data from everyday clinical practice in the same manner anywhere in the world, so outcomes across populations can be compared. This should ultimately improve patient safety by providing a snapshot of how new hemophilia drugs are being used and what adverse events are reported around the world, shedding light on new side effects and answering questions about products’ long-term safety throughout the population or in specific subgroups.

In addition to the sponsors scientific pursuits, this project furthers implementation of European Union rules on pharmacovigilance (Directives 2010/84/EU and 2012/26/UE) and the operating procedures for drug safety described in Italys Ministerial Decree of May 30, 2015. The rationale for the app is to strengthen the drug monitoring system of new hemophilia products that have been or will soon be authorized for sale, and to increase the involvement of patients and healthcare professionals in reporting adverse events to the authorities (Agenzia Italiana del Farmaco [AIFA] and the European Medicines Agency [EMA]).

Finally, an app for doctors and patients through which patients can fill in questionnaires about their health and physicians can check data and patient input will facilitate doctor-patient communication.