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Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study
Author(s) -
Oldenburg Johannes,
Shima Midori,
KruseJarres Rebecca,
Santagostino Elena,
Mahlangu Johnny,
Lehle Michaela,
Selak Bienz Nives,
Chebon Sammy,
Asikanius Elina,
Trask Peter,
Mancuso Maria Elisa,
JiménezYuste Victor,
Mackensen Sylvia,
Levy Gallia G.
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28474
Subject(s) - medicine , adverse effect , pediatrics , confidence interval , quality of life (healthcare) , bleed , regimen , surgery , nursing
Background Data regarding management of pediatric persons with hemophilia A (PwHA) with factor VIII (FVIII) inhibitors are limited. This prospective noninterventional study (NCT02476942) evaluated annualized bleeding rates (ABRs), safety, and health‐related quality of life (HRQoL) in pediatric PwHA with FVIII inhibitors. Procedure PwHA aged <12 years with current FVIII inhibitors and high‐titer inhibitor history were enrolled. Participants remained on usual treatment; no interventions were applied. Outcomes included ABR, safety, and HRQoL. Results Twenty‐four PwHA aged 2‐11 years (median 7.5) were enrolled and monitored for 8.7‐44.1 weeks (median 23.4). In the episodic (n = 10) and prophylactic (n = 14) groups, respectively, 121 of 185 (65.4%) and 101 of 186 (54.3%) bleeds were treated using activated prothrombin complex concentrate (aPCC) and/or recombinant activated FVII (rFVIIa). ABRs (95% confidence interval) were 19.4 (13.2‐28.4) and 18.5 (14.2‐24.0) for treated bleeds, and 32.7 (20.5‐52.2) and 33.1 (22.4‐48.9) for all bleeds, respectively. Most prophylactic group participants (92.9%) were prescribed aPCC; 50% adhered to their prescribed treatment regimen. Adherence to prophylactic rFVIIa was not assessed. Serious adverse events included hemarthrosis (12.5%) and mouth hemorrhage (12.5%); the most common nonserious adverse event was viral upper respiratory tract infection (12.5%). HRQoL showed functional impairment at baseline; scores remained stable throughout, with little intergroup variation. Conclusions ABRs remained high in pediatric PwHA with inhibitors receiving standard treatment. This study demonstrates the need for more effective treatments, with reduced treatment burden, to prevent bleeds, increase prophylaxis adherence, and improve patient outcomes.