Innovating rare disease care: A digital health network for the remote management of acquired Hemophilia A

Abstract Introduction Acquired Hemophilia A (AHA) is a rare but potentially life-threatening bleeding disorder caused by autoantibodies that neutralize coagulation factor VIII. The condition predominantly affects the elderly, due to its rarity, AHA often suffers from diagnostic delays and suboptimal treatment approaches, leading to increased morbidity and mortality risks as well as high costs for the healthcare system. Despite advances in available haemostatic treatments and immunosuppressive options, evidence on the long-term management of relapsed or refractory AHA remains furthermore limited. In this context, the current project aimed to design an integrated care network supported by a digital health platform. This system is able to connect peripheral hospitals, laboratories, community pharmacies, with the reference Hemophilia Center, enabling real-time, telematic management of AHA patients, from diagnostic suspicion to front line therapy and follow-up. Methods The project was centered around the creation of a digital platform able to facilitate remote clinical and patient interactions at multiple levels. Clinicians, pharmacists, biologists, clinical pathologists, and nurses are allowed to interact via this system to support diagnostic, therapeutic, and monitoring decisions. The platform allows data entry and sharing across all participating sites, ensuring the patient's clinical information is accessible in real time by specialists at the reference center as well as by local healthcare professionals. The digital platform has also a section specifically dedicated to patients to favor direct communication with therapists during follow-up. The digital infrastructure is designed to support: (1) hemophilia experts remote consultations from peripheral emergency departments, medical units and general practitioners; (2) centralized interpretation of laboratory assays, including standard coagulation tests and FVIII activity; (3) definition of front line therapy and following therapeutic adjustments based on real-time clinical and laboratory data. An important aspect of the platform is the protection of patient privacy. All data are protected in compliance with applicable privacy regulations and security protocols, ensuring the confidentiality and integrity of patient information. Results On December 6, 2023, the Hemophilia Reference Regional Center of Palermo started to develop the digital platform, which became operational and in use across all the region in February 2025. To date, four physicians from four community hospitals have joined the network, two from emergency departments (ER) and two from haematology units (HU); two AHA patients have been managed with this integrated system, one from an ER and another from HU. Twelve consults with the haemophilia Center have been asked through the platform for AHA suspicion. After hospital discharge, patients are currently regularly monitored, and laboratory and clinical data are entered by their general practitioners. Using this integrated digital system, therapy has been remotely tailored to the patients' needs, leading to AHA complete remission in both cases. Six months after its availability the platform underscores its potential to streamline care, reduce inappropriate interventions, and enhance communication across all levels of the healthcare systems. Conclusions This digital platform could serve as a blueprint for managing other rare diseases by bridging gaps between specialized centers and peripheral care providers, fostering innovation and systemic improvement in rare disease care delivery. The integration of clinical expertise, real-time data exchange, and patient engagement has the potential to significantly improve diagnosis, treatment, and long-term monitoring of AHA patients. Furthermore, the platform enhances the capacity of the Italian National Health System to efficiently manage rare and complex diseases. The model supports a patient-centered approach that ensures equitable access to care throughout the region.