VATIS UPDATE Article

Aiming to find ways to implement viable, practical and sustainable haemophilia care strategies in the country, the Department of Biotechnology (DBT), under the Ministry of Science and Technology, government of India, will soon begin research in the area of haemophilia which is an X-linked hereditary single gene bleeding disorder, leading to deficiency of coagulation factor VIII (FVIII) or factor IX (FIX). It manifests as spontaneous or trauma-induced haemorrhagic episodes in patients, progressing to chronic disabilities like arthropathy & contractures and premature mortality in untreated patients or patients with sub-optimal treatment.

Priority areas for research include novel approaches to diagnosis of haemophilia; development of recombinant factors or novel therapies; development of new viral inactivation/removal technologies for plasma products; development of novel approaches to Immune Tolerance Induction therapy (ITI) to treat inhibitors that may develop during CFC replacement treatment of haemophilia; genetic evaluation of the full spectrum of hereditary bleeding disorders which are common in the country to help design a comprehensive cost effective diagnostic strategy; and strategies for development of gene therapy approaches for haemophilia.