Background:

Hemophilia, an X-linked recessive bleeding disorder, presents significant challenges in management but can be mitigated with appropriate clotting factor replacement therapy. Resource limited settings across the world face unique challenges in implementing prophylaxis as the standard of care. The objective of this study is to assess the improvement in prophylaxis and comprehensive care of children with Hemophilia(CwH) attached to a newly established Hemophilia Treatment Center (HTC) in India during the last decade.

Methodology:

Study was retrospective from January 2015 to June 2024. The HTC started functioning in a Government District Hospital in 2014. It had access to an accredited blood center. A coagulation laboratory doing factor and Bethesda assays was established and accredited. Educational sessions were conducted for patients , caregivers and healthcare workers. Fortnightly comprehensive care clinics were conducted with Hematologist , Paediatrician, Physiatrist , Physiotherapist and Laboratory Technologist. Cost of treatment was borne by local self-government and state. All patients <18 years with Hemophilia A and B registered in the center were included for analysis . Those staying near the center (< 50 km) were evaluated for access to prophylaxis. Clotting factor concentrate(CFC) are plasma-derived or recombinant CFCs and it provides convenient high doses of clotting factor for the treatment and prevention of bleeds. Standard half life CFCs need frequent venipunctures for prophylaxis as it has short half life when compared to Extended half life CFCs. Emicizumab is a chimeric bispecific antibody directed against the enzyme activated Factor IX and the zymogen FX that mimics the co-factor function of Factor VIII in patients with hemophilia A, with or without inhibitors. Proportion of adults and children on prophylaxis , age at initiation of prophylaxis , Annual bleed rates (ABR), Functional Independence Score in Hemophilia(FISH), Hemophilia Joint Health Score(HJHS) and Prevalence of inhibitors were measured. Patients on prophylaxis were regularly reviewed and treatment changes made to keep annual bleed rates 0-3. Outcomes of 2024 were compared against 2015.

We used longitudinal mixed effects Poisson regression with a random intercept for patients and a fixed effect for time to model trends in the bleeding rate. Quantitative patient characteristics are summarised by means and standard deviations and categorical patient characteristics are summarized by frequencies and percentages. p values <0.05 is considered as statistically significant. Analysis were performed in R version 4.4.1.

Results:

Patients with Hemophilia(PwH) resgistered in the HTC were 441 in 2015 and increased to 874 in 2024, out of which 734(84%) had Hemophilia A(HA) ; 726(83.1%) had severe disease.

Children with Hemophilia (CwH)in 2024 was 281(38.7%) against 128(38.3%) in 2015. CwH living in close proximity and eligible for prophylaxis from center were 37. No CwH were on prophylaxis in January 2015 and 32(86.5%) CwH received Prophylaxis in 2024. CFC use was 1894 IU/kg/year/person and 46.57 mg/kg/year/person for Emicizumab. ABR of 1.13(p=0.006) in 2024 vs 11 in 2015 and over the years, ABR has decreased significantly by a rate ratio of 0.8(95% CI: 0.76-0.85, p <0.001).The mean HJHS score was 1.43 (SD 2.06). There was a significant decrease in the average HJHS score per year by 0.84(95% CI: 0.71 - 1, p= 0.05). The mean FISH score was 31.7(SD 1.09) and FISH score did not change significantly over the years(p=0.83). Proportion of adults receiving prophylaxis were 7(12.7%).

Conclusion: The establishment of HTC and its comprehensive approach, including prophylaxis, education, and financial support, has substantially improved the care of CwH in a resource-limited setting. Increased prophylaxis coverage, reduced ABR, improved HJHS and maintained FISH scores reflect improvement in care. Enhanced access to newer treatments like Emicizumab reflect significant progress in patient management and outcomes over the past decade. Limitations are retrospective, single center study.

Disclosures

No relevant conflicts of interest to declare.

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2024
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