Continuum of Care for Hemophilia: The Story of India

Hemophilia A and hemophilia B are disorders of coagulation caused by deficiency of clotting factors VIII and IX respectively. Genes for both these factors are on X chromosome and are prototypes of X-linked recessive disorders, manifesting only in males. Taken together, they are one of the commonest monogenic disorders in the world and appear to be equally prevalent in India. Diagnosis and treatments have been available for decades and have evolved over time. The story of successful gene therapy is as important as the dramatic changes occurring in the quality of life of hemophilia patients in India with availability of coagulant factors free of cost with the support of government funds. New drugs like emicizumab, which is a long acting non-factor agent, has made prophylaxis easy with one subcutaneous injection per month; the patient can forget that he has hemophilia. Other novel drugs acting downstream of the factor VIII and IX in the coagulation cascade like antithrombin antibody and anti-tissue factor pathway inhibitor monoclonal antibody, have shown great promise in clinical trials. With approval to gene therapies for both, hemophilia A and B, the persons with hemophilia are going to have great choices of therapies on their platter. It is a happy time that these therapies are likely to be within reach of the patients in India; thanks to the patient support group and the will of the policy makers.