Patient Reported Outcomes to Assess Quality of Hemophilia Care in North India - Results of a Global Partnership
Authors: Jain Akshat , Mehta Sudhir , Joshi Mrinal , Garg Kapil , Goyal Laxmi Kant , Parnes Aric , Al-Sayegh Hasan , Ma Clement , Neufeld Ellis J. 
Published: October 2019

Abstract
Deficiency of clotting factors VIII (Hemophilia A) and IX (Hemophilia B) represent one of the most debilitating inherited groups of bleeding disorders. According to the most recent survey report from the World Hemophilia Federation, an estimated 178,500 patients suffer from hemophilia globally (143,000 Hemophilia A, 28,000 with Hemophilia B approximately). By most recent estimates from 2014 India has surpassed every country in the global database with a total of 17,470 patients with hemophilia ahead of USA with its 17,131 reported cases. Infectious disease and perinatal mortality take precedence for resource allocation in evolving economies like India, presenting an ongoing challenge for "rare" diseases like hemophilia.
This study assesses the quality of hemophilia care at the SMS Medical School which is the flagship medical care center in the northwestern Indian city of Jaipur, the capital of the largest Indian state with an area of 0.34 million square kilometers and population of 68.55 million. Since the introduction of the free factor distribution in the fall of 2012, the program grew from a modest patient population size of 60 patients to a robust 700 plus patients within 4 years aided by the World Hemophilia Twinning program grant. A standardized self-administered questionnaire was administered to all the hemophilia patients seen at the outpatient comprehensive hemophilia program (CHP) at the SMS Medical College between February 1, 2016 and May 5, 2016.
Two hundred patients met the inclusion criteria and participated in the study. One hundred eighty-seven (94%) and 13 (6) patients were diagnosed with hemophilia A and B respectively; 126 (63) had severe disease while 65 (33) had moderate and 9 (5) had mild hemophilia. In an expected male predominant sample (n=198 males [99.5]), the median age at the time of study was 12 years (range=1-53 years), and 144 (72) patients were from rural outskirts of the resource poor state visiting the tertiary care center for hemophilia care. Contrary to popular belief, only a minority 11% and 1% of patients identified themselves of the Muslim and Sindhi faith respectively, versus a majority 87% who followed Hinduism. Tenets of consanguinity and family size have often led to misunderstanding and stigmatization of the disease in the Asian subcontinent prior to this study thus far. Despite a majority 63% patients suffering from severe hemophilia, nearly half (44%) reported a delay of more than 6 months in diagnosis time from the first bleed. Remarkably, 96 % reported to know their diagnosis fully and 93% reported understanding that hemophilia is a genetically transmitted disorder but approximately 82.5 % did not know if they ever underwent testing for viral infections (H.I.V, Hepatitis B, C ) since their diagnosis . Nearly 45 % were offered genetic counseling services at some point during their care, a remarkable feat for a hemophilia program in a resource strapped environment. Inpatient stay for bleeds and complications in this predominantly severe mix of patients was encouragingly less than 1-5 days in 93% of the patients but despite free drug delivery program 79% families reported an out of pocket expense of more than 10,000 Indian rupees (INR; approximately 147 USD) during the hospital stay. This in a state where 65% of patients reported per capital household income less than 100,000 INR (1,485 USD) was particularly concerning. Nearly 18 % of the respondents identified themselves as below poverty (BPL) and enjoyed the benefits of free transportation under the governmental subsidies through a BPL card. Household income and below poverty status were not associated with hemophilia care outcomes (p-values <0.1).
Reported favorable patient satisfaction (86%) and continued access to free factor infusion services irrespective of socio economic status as demonstrated by this study, for a resource limited setting caring for over 600 hemophilia patients should be considered a success. New epidemiologic insights from this study will help researchers and clinicians alike to design care delivery models for hemophilia. Not only does this study reflect the power of international collaborations in remarkably improving services for such ignored debilitating diseases in the resource limited nations but also presents as a unique first quality assessment tool for the nation with the largest number of identified hemophilia patients in the world.

Disclosures
Parnes: Pfizer: Consultancy; Baxalta: Research Funding. Neufeld:Pfizer: Consultancy; baxalta: Consultancy, Research Funding.