AHEAD International and German Studies: Subgroup Analysis of Patients with Moderate or Severe Hemophilia A Receiving Antihemophilic Factor (Recombinant) as Immune Tolerance Induction (ITI) Therapy
ISTH Academy. Ozelo M. Jul 9, 2019; 264620; PB1430 Topic: Hemophilia - Clinical
Margareth Ozelo
Margareth Ozelo
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PB1430

AHEAD International and German Studies: Subgroup Analysis of Patients with Moderate or Severe Hemophilia A Receiving Antihemophilic Factor (Recombinant) as Immune Tolerance Induction (ITI) Therapy

M.C. Ozelo1, K. Kurnik2, G. Giuffrida3, H. Jiang4, G.R. Chiu5, L. Renault6, A. Fernandez6, K. Khair7
1Hemocentro UNICAMP, University of Campinas, Campinas, São Paulo, Brazil, 2Dr. von Haunersches Kinderspital Klinikum der Universität München, München, Germany, 3Centro di Riferimento Regionale per Emofilia e Trombosi Modulo Dipartimentale Emofilia, Catania, Italy, 4Baxalta US Inc., a Takeda company, Cambridge, MA, United States, 5Shire US Inc., a Takeda company, Lexington, MA, United States, 6Shire International GmbH, a Takeda company, Zug, Switzerland, 7Centre for Outcomes and Experience Research in Children's Health, Illness and Disability (ORCHID), Great Ormond Street Hospital for Children, London, United Kingdom

Main Topic: Hemophilia and Bleeding (including Transfusion)
Category: Hemophilia - Clinical

Background: ITI therapy is the standard of care to restore FVIII pharmacokinetic/clinical response in patients with hemophilia A and FVIII inhibitors. The German (DRKS 00000556, started 2010) and international (NCT02078427; started 2011) Antihemophilic factor (recombinant) (rAHF) Hemophilia A outcome Database (AHEAD) studies evaluate long-term, real-world outcomes in patients with severe/moderate hemophilia A treated with rAHF (on-demand/prophylaxis/ITI).
Aims: To analyze outcomes in patients receiving rAHF ITI in these studies.
Methods: Study design and interim effectiveness/safety results from these non-interventional, prospective, multicenter, cohort studies with ethics-committee approval and patient informed-consent were previously reported (Ozelo et al, Haemophilia 2019;25[suppl 1];P186). In this analysis, ITI outcomes were defined as success (complete [CR] or partial [PR] response) or failure based on FVIII inhibitor reversal in absence of available pharmacokinetic assessments. Average daily rAHF ITI doses were calculated from weekly doses because patient treatment schedules varied. Data cutoff: 13 April 2018.
Results: Of overall 1099 enrolled AHEAD patients, 22 received rAHF ITI. 15 patients had complete 5-year follow-up/ITI outcomes data; median (range) age at enrollment was 9 (1-44) years. 12/15 patients had severe, 3/15 had moderate disease. Median (range) historic FVIII inhibitor peak: 11.9 (3-288.5) BU. Median (range) daily rAHF dose: 10.2 (4.8-135.0) IU/kg. CR and PR were achieved by 7 patients each; median (range) time to reach CR/PR: 20.5 (9.3-26) months. Patients achieving CR were younger (median [range] years, 3 [1-13] vs 13 [4-44]) and received higher rAHF doses (median [range] IU/kg/day, 63.5 [4.8-135.0] vs 8.6 [5.9-57.1]) than patients achieving PR. Response failure occurred in 1 patient (age at enrollment: 34 years; historic FVIII peak: 148 BU; daily rAHF dose: 9 IU/kg).
Conclusions: Preliminary results in this small number of patients receiving rAHF ITI therapy in a real-world setting indicate effectiveness of this treatment modality, with 14/15 patients achieving CR or PR.

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