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Impact of Extended Half-life Product versus Conventional Factor Product in Children with Hemophilia A – Findings from a Tertiary Care Centre

1King Faisal Specialist Hospital & Research Center, Pediatric Hematology / Oncology, Riyadh, Saudi Arabia, 2King Faisal Specialist Hospital & Research Center, Oncology Center Department, Riyadh, Saudi Arabia, 3King Faisal Specialist Hospital & Research Center, Pathology, Riyadh, Saudi Arabia, 4Princess Noura University, Riyadh, Saudi Arabia, 5King Faisal Specialist Hospital & Research Center, Pediatric Nursing, Riyadh, Saudi Arabia

Abstract Number: PB1059

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Until 2014, available FVIII products had Short Half-life (SHL) requiring frequent intravenous injections; FDA approved the first Extended Half-life (EHL) product known as Eloctate (rFVIII Fc). Pediatric hematology section at King Faisal Specialist Hospital and Research Centre, Riyadh, is a tertiary referral center for clotting factor deficiencies.

Aims: To report our experience with EHL products usage in pediatric (≤14 years) patients: Annual Bleeding Rate (ABR), Annual Factor Consumption (AFC) and compare bleed-free survival between EHL versus SHL products.

Methods: .Data collected on REDCap (Research Electronic Data Capture) software and analysis performed utilizing SPSS Version 20.0 software.

Results: Eloctate introduced to the institutional formulary on November 25th, 2017; from 107 HA cases in the pediatric service, 60 (58%) children (≤14 years) switched to Eloctate while 47 children remains on SHL products. Lesser infusion the main (67%) reason to switch followed by latest technology (18%) and 15 % (9) switched to receive Eloctate Immune Tolerance Therapy (ITI) protocol. Prophylactic treatment observed in 67% (40) pre-switch, while 98% (59) are on prophylactic treatment.AFC was calculated for 46 patients who completed ≥ 12 months post-switch follow-up: 50% reduction in AFC, while bleed-free survival was calculated in both groups with 80% (48/60) children on EHL are bleed-free as compared to 59% (124/47) in SHL group; p-value = 0.074, longer follow-up warranted. Cost-effective analysis demonstrated reduction in bleeding -related hospital visits and admissions cost for EHL group, a detailed analysis is underway.

Conclusions: In our experience patients and families are aware and keen on adapting EHL products, findings suggest improved bleed-free survival and reduced AFC. Structured QoL survey would enable patient satisfaction levels between two groups. Preliminary financial assessments underlines cost-effectiveness of adapting EHL products but longer follow-up would enable a model (Markov) for countries in the region planning to integrate EHL in respective National hemophilia care programs.

To cite this abstract in AMA style:

Saleh M, Alzahrani H, Owaidah T, Aljefri A, Mohammed D, Safi B, Mohammed V. Impact of Extended Half-life Product versus Conventional Factor Product in Children with Hemophilia A – Findings from a Tertiary Care Centre [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/impact-of-extended-half-life-product-versus-conventional-factor-product-in-children-with-hemophilia-a-findings-from-a-tertiary-care-centre/. Accessed October 1, 2023.

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