Evidence map›Paper›PMID 41636686›Full record

Observational studyJournal of managed care & specialty pharmacy2026

Real-world adherence to and persistence with emicizumab in people with hemophilia A using a national claims database.

Janet S Lee, Robert Schuldt, Zhiyu Xia, Lucy Lee, Juliana M L Biondo, Amy D Shapiro

Abstract readObservational Study
In one paragraph

Observational study in Journal of managed care & specialty pharmacy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0citing papers in PubMed
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1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Janet S LeeGenentech, Inc., South San Francisco, CA.
Robert SchuldtGenentech, Inc., South San Francisco, CA.
Zhiyu XiaGenentech, Inc., South San Francisco, CA.
Lucy LeeGenentech, Inc., South San Francisco, CA.
Juliana M L BiondoGenentech, Inc., South San Francisco, CA.
Amy D ShapiroIndiana Hemophilia & Thrombosis Center, Indianapolis.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundSubstantial rates of nonadherence are observed among people with hemophilia A (PwHA) receiving standard- or extended-half-life factor (F)VIII replacement products, although better adherence is associated with fewer bleeding events and better quality of life. Emicizumab is the first bispecific FIXa-/FX-directed antibody approved for routine prophylaxis in PwHA. More real-world adherence and persistence data on emicizumab and other new hemophilia treatments are needed to better inform decisions on population health care and individualized treatments for PwHA.

objectiveTo describe real-world emicizumab adherence and persistence among PwHA in the United States.

methodsThis retrospective, observational cohort study used adjudicated health plan claims data from IQVIA PharMetrics Plus. This study included PwHA with at least 1 claim noting hemophilia A diagnosis between May 1, 2017, and September 30, 2023; at least 2 claims for emicizumab fills between November 1, 2017, and September 30, 2023; and continuous medical and pharmacy insurance benefit coverage for at least 6 months before and at least 12 months after the first emicizumab fill (index date). Adherence to emicizumab was measured during the 12-month post-index period using the proportion of days covered (PDC). Persistence was defined as the proportion of PwHA who did not discontinue emicizumab during the post-index period, where discontinuation was defined as a treatment gap of at least 60 days. Among adherent PwHA (PDC ≥ 80%), the number of post-index claims for FVIII were evaluated.

resultsA total of 280 PwHA were included in the study, of whom 98% were male and the mean age was 26 (SD = 16) years. Overall, 76% of PwHA were adherent to and 85% of PwHA were persistent with emicizumab. Rates of adherence and persistence were high across age- and geography-based subgroups, ranging from 61% to 97%. Among PwHA adherent to emicizumab (n = 213), 57% had no post-index claims for FVIII.

conclusionsIn the first year following emicizumab initiation, high adherence and low discontinuation rates were observed among PwHA in a real-world setting.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedDrug MonitoringHemophilia AAdolescentAdultChildChild, PreschoolCohort StudiesDatabases, FactualFactor VIIIFemaleHumansMaleMiddle AgedRetrospective StudiesAntibodies, BispecificAntibodies, Monoclonal, HumanizedemicizumabFactor VIII

Identifiers

PMID41636686
PMCPMC12871514

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.