Outcomes in children with hemophilia A with inhibitors
Standard
Outcomes in children with hemophilia A with inhibitors : Results from a noninterventional study. / Oldenburg, Johannes; Shima, Midori; Kruse-Jarres, Rebecca; Santagostino, Elena; Mahlangu, Johnny; Lehle, Michaela; Selak Bienz, Nives; Chebon, Sammy; Asikanius, Elina; Trask, Peter; Mancuso, Maria Elisa; Jiménez-Yuste, Victor; von Mackensen, Sylvia; Levy, Gallia G.
In: PEDIATR BLOOD CANCER, Vol. 67, No. 10, 10.2020, p. e28474.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
Harvard
APA
Vancouver
Bibtex
}
RIS
TY - JOUR
T1 - Outcomes in children with hemophilia A with inhibitors
T2 - Results from a noninterventional study
AU - Oldenburg, Johannes
AU - Shima, Midori
AU - Kruse-Jarres, Rebecca
AU - Santagostino, Elena
AU - Mahlangu, Johnny
AU - Lehle, Michaela
AU - Selak Bienz, Nives
AU - Chebon, Sammy
AU - Asikanius, Elina
AU - Trask, Peter
AU - Mancuso, Maria Elisa
AU - Jiménez-Yuste, Victor
AU - von Mackensen, Sylvia
AU - Levy, Gallia G
N1 - © 2020 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.
PY - 2020/10
Y1 - 2020/10
N2 - 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.
AB - 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.
KW - Antibodies, Bispecific/therapeutic use
KW - Antibodies, Monoclonal, Humanized/therapeutic use
KW - Child
KW - Child, Preschool
KW - Factor VIII/antagonists & inhibitors
KW - Female
KW - Follow-Up Studies
KW - Hemophilia A/drug therapy
KW - Humans
KW - Infant
KW - Male
KW - Prospective Studies
KW - Quality of Life
KW - Retrospective Studies
KW - Treatment Outcome
U2 - 10.1002/pbc.28474
DO - 10.1002/pbc.28474
M3 - SCORING: Journal article
C2 - 32776489
VL - 67
SP - e28474
JO - PEDIATR BLOOD CANCER
JF - PEDIATR BLOOD CANCER
SN - 1545-5009
IS - 10
ER -
