Bleeding events in people with hemophilia B on prophylactic factor IX treatment: a systematic review and meta-analysis.
7 affiliations
- 1Evidence Synthesis, IQVIA, Falls Church, Virginia, USA.
- 2Evidence Synthesis, IQVIA, San Mateo, California, USA.
- 3Pfizer, New York City, New York, USA.
- 4Pfizer, Montreal, Quebec, Canada.
- 5Pfizer, Collegeville, Pennsylvania, USA.
- 6Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA.
- 7Hemostasis and Thrombosis Center, Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, California, USA.
Abstract
Background
Hemophilia B is a rare, X-linked disorder leading to deficient or dysfunctional clotting factor IX and bleeding complications.Objectives
The objective of this systematic review and meta-analysis was to summarize bleeding events in people with hemophilia B of any severity without inhibitors receiving prophylactic factor IX replacement therapies to better understand disease burden and unmet needs.Methods
A systematic search was conducted to identify clinical trials and observational studies reporting annualized bleeding rate (ABR), annualized joint bleeding rate, spontaneous bleeding events, traumatic bleeding events, and percentage of patients with zero-bleeding events. Bleeding outcomes were specified as total, treated, or unspecified and analyzed using the random-effects model. Subgroup meta-analyses assessed differences by age, treatment type, follow-up duration, and publication date, when feasible.Results
The search yielded 5684 citations; 24 clinical trials and 53 observational studies were included in the meta-analysis. Treated ABR was 2.23 (95% CI, 1.52-3.27) in 13 trials; treated or unspecified ABR was 2.25 (95% CI, 1.67-3.03) in 22 trials. In 48 observational studies, treated or unspecified ABR was 2.08 (95% CI, 1.62-2.66). ABR was generally lower for patients <12 years of age (vs ≥12 years of age) and patients treated with extended half-life (vs standard half-life).Conclusion
Results from this meta-analysis reflect advances in hemophilia B treatment, but also highlight unmet needs, with many patients experiencing ≥2bleeding events per year. Inconsistent ABR definitions and variability in follow-up times may introduce heterogeneity in the combined estimates.
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BibTeX
@article{lindsley2026bleeding,
title = {{Bleeding events in people with hemophilia B on prophylactic factor IX treatment: a systematic review and meta-analysis.}},
author = {Kristina Lindsley and Arianna Nevo and Carmine Colavecchia and Lisa J Wilcox and Priya Patel and Carlos Estevez and Cynthia Khanji and Jennifer Uyei and Steven W Pipe and Guy Young},
journal = {Research and practice in thrombosis and haemostasis},
year = {2026},
month = sep,
doi = {10.1016/j.rpth.2026.106954},
url = {https://europepmc.org/article/MED/42910129},
note = {CC BY},
}
RIS
TY - JOUR
TI - Bleeding events in people with hemophilia B on prophylactic factor IX treatment: a systematic review and meta-analysis.
AU - Kristina Lindsley
AU - Arianna Nevo
AU - Carmine Colavecchia
AU - Lisa J Wilcox
AU - Priya Patel
AU - Carlos Estevez
AU - Cynthia Khanji
AU - Jennifer Uyei
AU - Steven W Pipe
AU - Guy Young
PY - 2026
DA - 2026/09/09
JO - Research and practice in thrombosis and haemostasis
DO - 10.1016/j.rpth.2026.106954
UR - https://europepmc.org/article/MED/42910129
AB - Background Hemophilia B is a rare, X-linked disorder leading to deficient or dysfunctional clotting factor IX and bleeding complications.Objectives The objective of this systematic review and meta-analysis was to summarize bleeding events in people with hemophilia B of any severity without inhibitors receiving prophylactic factor IX replacement therapies to better understand disease burden and unmet needs.Methods A systematic search was conducted to identify clinical trials and observational studies reporting annualized bleeding rate (ABR), annualized joint bleeding rate, spontaneous bleeding events, traumatic bleeding events, and percentage of patients with zero-bleeding events. Bleeding outcomes were specified as total, treated, or unspecified and analyzed using the random-effects model. Subgroup meta-analyses assessed differences by age, treatment type, follow-up duration, and publication date, when feasible.Results The search yielded 5684 citations; 24 clinical trials and 53 observational studies were included in the meta-analysis. Treated ABR was 2.23 (95% CI, 1.52-3.27) in 13 trials; treated or unspecified ABR was 2.25 (95% CI, 1.67-3.03) in 22 trials. In 48 observational studies, treated or unspecified ABR was 2.08 (95% CI, 1.62-2.66). ABR was generally lower for patients <12 years of age (vs ≥12 years of age) and patients treated with extended half-life (vs standard half-life).Conclusion Results from this meta-analysis reflect advances in hemophilia B treatment, but also highlight unmet needs, with many patients experiencing ≥2bleeding events per year. Inconsistent ABR definitions and variability in follow-up times may introduce heterogeneity in the combined estimates.
SN - 2475-0379
ER -