Management of Hemophilia in Patients with Inhibitors: The Perspective from Developing Countries

Abstract

Data are limited on inhibitors in people with hemophilia (PWH) in developing countries. There is a perception that the overall prevalence of inhibitors, ranging from 7 to 19% in different reports, may be lower in these countries as compared with that reported from developed countries. This is possible given the fact that most patients are treated after 2 years of age with plasma-derived clotting factor concentrates. Whether genetic or other environmental factors also contribute to this needs further evaluation. There is a need to develop laboratory infrastructure and establish quality control programs for laboratory tests for inhibitors in developing countries. Management options vary widely given the socioeconomic diversity among these countries. Significant individualization of approach to management is therefore required depending on the available resources, particularly with regard to the use of bypassing agents. The limited data on immune tolerance induction with some low-dose regimens deserve further evaluation. Even in resource-constrained environments, education and a policy of systematic screening of patients associated with judicious use of bypassing agents can significantly improve the care of PWH who develop inhibitors.

Clinico-epidemiological profile of hemophilia with special reference to development of inhibitors in Indian scenario: a crosses sectional study at tertiary care centre in north India

“…However, ITT is still not feasible in most centres in India because of prohibitive cost and logistics constraints. 4 Other therapies available are High-dose factor concentrates, bypassing agents and immunoadsorption. In the case of failure of bypassing agents to control bleeding, immunoadsorption may temporarily reduce the inhibitor titre in high-responder inhibitor patients, enabling effective replacement therapy with factor concentrates.…”

Hemophilia Care in India: A Review and Experience from a Tertiary Care Centre in Uttar Pradesh

“…The prevalence of inhibitors in India is estimated to be 8.2-13% [13,14]. During a 5 year follow up, 23 of 283 severe hemophilia patients were investigated for the presence of inhibitors before surgery or because of a suboptimal response to factor replacement therapy [15].…”

“…Diagnosing inhibitors, which are usually immunoglobulins (Ig) of the IgG class, demands specialized laboratories and expertise [14,21,22]. Each hemophilia treatment center should have its own protocol for the detection of inhibitors, and this protocol should involve systematic assessment, regardless of whether it is based on the number of exposure days, conducted before any surgical procedure, or in if it is conducted in case there is no response to regular factor replacement therapy [14].…”

“…The ultimate goal of treating PWH and inhibitors is to permanently eradicate inhibitors via immune tolerance therapy (ITT), and the same therapy has been indicated for high-responder patients in developed countries [26]. However, ITT is still not feasible in most centers in India because of prohibitive cost and logistics constraints [14].…”

“…Low titers or low response patients could be managed with higher doses of factor concentrates to overcome inhibitor levels [14] during mild to moderate bleeds. On-demand therapy with bypassing agents is the first option for high titer or high response patients.…”