Low-dose immune tolerance induction therapy in children of Arab descent with severe haemophilia A, high inhibitor titres and poor prognostic factors for immune tolerance induction treatment success.

Autor: Elalfy M; Hemophilia Care Centre, Faculty of Medicine, Ain Shams University, Cairo, Egypt., Elghamry I; Hemophilia Care Centre, Faculty of Medicine, Ain Shams University, Cairo, Egypt., Hassab H; Hemophilia Care Centre, Faculty of Medicine, Alexandria University, Alexandria, Egypt., Elalfy O; National Research Centre, Complementary Medicine, Cairo, Egypt., Andrawes N; Hemophilia Care Centre, Faculty of Medicine, Ain Shams University, Cairo, Egypt., El-Ekiaby M; Hematology Unit, Blood Transfusion Centre, Shabrawishi Hospital, Giza, Egypt.
Jazyk: angličtina
Zdroj: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2022 Jan; Vol. 28 (1), pp. 65-72. Date of Electronic Publication: 2021 Nov 19.
DOI: 10.1111/hae.14456
Abstrakt: Introduction: Immune Tolerance Induction (ITI) is the first-choice therapy to eradicate Factor VIII (FVIII) neutralizing antibodies in patients with haemophilia A (HA). There is limited published data on ITI from East Mediterranean countries.
Aim: To assess the effectiveness of a low-dose ITI regimen to eradicate FVIII neutralizing antibodies in children with severe HA and high-titre inhibitors.
Methods: A prospective, single-arm study was conducted in children with HA (FVIII < 1 IU/dl), high-titre inhibitors and poor prognostic factors for successful ITI. Patients were treated with ∼50 IU/kg plasma-derived FVIII containing von Willebrand factor (pdFVIII/VWF) concentrate (Koate-DVI, Grifols) three times a week. Time to achieve tolerance, total and partial success were analysed after ITI. Annual bleeding rate (ABR), number of target joints, FVIII recovery and school absence were compared before and after ITI.
Results: Twenty patients with median (range) age of 6.2 (3-12) years and pre-ITI inhibitor titre of 36.5 (12-169) BU were enrolled. ITI lasted ≤12 months (early tolerization) in 45% of patients. Median follow-up was 12 months (3-22) and total response rate was 80% (60% total success; 20% partial success). Patients with two and three poor prognosis factors achieved overall success rate of 60% and 50%, respectively. ABR, target joints and school absence were reduced after ITI by 60%, 50% and 44.1%, respectively. In successful ITI tolerized patients, FVIII recovery was 90 (60-100)%.
Conclusion: A low-dose ITI therapy using a pdFVIII/VWF concentrate achieved at least partial tolerance in 80% of patients, and reduced annual bleeds in children with high inhibitor titres and at least one poor prognosis factor for ITI treatment success.
(© 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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