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Treatment of Hemophilia A with rVIII-SingleChain in Italy: Physician and Patient Survey

1Centro di Riferimento Regionale Malattie Emorragiche e Trombotiche dell'Adulto Ematologia U, Città della Salute e della Scienza, Turin, Italy, 2Centro per le Malattie Emorragiche e della Coagulazione, AOU Careggi, Florence, Italy, 3Immunologia e Medicina Trasfusionale, Ospedale S. Luca, Vallo della Lucania - Salerno, Italy, 4Ematologia, AOU Policlinico Umberto I, Rome, Italy, 5Dipartimento di Scienze Biomediche e Oncologia Umana - Unità di Pediatria, Università degli Studi di Bari 'Aldo Moro', Bari, Italy, 6U.O. di Onco-Ematologia, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy, 7UOSD Emofilia e Trombosi, AOU Consorziale Policlinico di Bari, Bari, Italy, 8UOC Genetica Medica - Dipartimento di Medicina Clinica e Sperimentale, Università di Foggia, Foggia, Italy, 9Centro Regionale di Riferimento per le Malattie Emorragiche, Ospedale Giannina Gaslini, Genoa, Italy, 10Centro di Riferimento Regionale Malattie Emorragiche e Trombotiche Ereditarie in Età Pediatrica, AOU Città della Salute e della Scienza - Ospedale Infantile Regina Margherita, Turin, Italy, 11Hospital S.M. di Loreto Nuovo, Naples, Italy, 12Centro Emofilia e Trombosi A. Bianchi Bonomi, Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Milan, Italy, 13Unità Emofilia, Emostasi e Trombosi, Azienda Ospedaliera Pugliese Ciaccio, Catanzaro, Italy, 14Centro di Riferimento Regionale per le Emocoagulopatie, AORN Santobono Pausilipon, Naples, Italy, 15Centro Emofilia, AOU di Padova, Padua, Italy

Abstract Number: PB1050

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: rVIII-SingleChain is indicated for treatment and prophylaxis of bleeding in patients with hemophilia A (HA). The safety and efficacy of rVIII‑SingleChain have previously been shown in the AFFINITY clinical trial program.

Aims: This survey evaluated clinical experience with rVIII‑SingleChain from the perspective of both physicians and patients.

Methods: A web-based survey (July-September 2019) involving 14 Hemophilia Treatment Centers (HTCs) collected physician-reported data about HA patients receiving rVIII-SingleChain for ≥12 months. Additionally, a number of patients were interviewed.

Results: In total, 48 patients received rVIII-SingleChain for ≥12 months across the 14 HTCs; 38% were ≤18 years, 37% 19-40 years and 25% ≥41 years. Overall, 73% of patients had severe HA and 85% were receiving prophylaxis. Interviews were conducted with 26 patients; 38% were ≤18 years, 39% 19-40 years and 23% ≥41 years, 62% had severe HA and 85% were receiving prophylaxis. For patients who had received prophylaxis with a prior FVIII product, the median dosing frequency reduced from 3 to 2 infusions per week following the switch to rVIII-SingleChain. The annual median factor consumption was 4,500 IU/kg (range 2,215-7,200 IU/kg) with prior product and 4,320 IU/kg (range 2,215-6,646 IU/kg) with rVIII-SingleChain. Both physicians and patients reported a reduction in annualized spontaneous bleeding rate with rVIII‑SingleChain compared with prior product (mean 2.15 vs. 0.89 and 2.46 vs. 0.63 events/year; median: 1.0 vs. 0.0 and 2.0 vs. 0.0, respectively). Mean satisfaction ratings (from 1; dissatisfied, to 5; very satisfied) for rVIII-SingleChain were quite high both for physicians (4.14, 86% satisfied/very satisfied) and for patients (4.18, 87% satisfied/very satisfied).

Conclusions: This survey suggested that switching to rVIII-SingleChain allowed patients to reduce their dosing frequency without increasing factor consumption or compromising protection. Both physicians and patients reported a positive experience with rVIII-SingleChain after at least 1 year of treatment.

To cite this abstract in AMA style:

Borchiellini A, Castaman G, Feola G, Ferretti A, Giordano P, Luciani M, Malcangi G, Margaglione M, Molinari AC, Pollio B, Rocino A, Santagostino E, Santoro C, Santoro R, Schiavulli M, Zanon E. Treatment of Hemophilia A with rVIII-SingleChain in Italy: Physician and Patient Survey [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/treatment-of-hemophilia-a-with-rviii-singlechain-in-italy-physician-and-patient-survey/. Accessed October 2, 2023.

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