A novel supplemental approach to capturing post-marketing safety information on recombinant factor VIIa in acquired hemophilia: the Acquired Hemophilia Surveillance project

Lentz, Steven R.; Tandra, Anand; Gut, Robert; Cooper, David L.

doi:10.2147/jbm.s55216

Cited by 6 publications

(12 citation statements)

References 2 publications

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“…The subset of acquired hemophilia-participating sites were predominantly adult HTCs within the HTRS registry, which at one time included up to 100 of the 141 federally designated HTCs in the United States [ 24 ]; however, at present, the United States has approximately 5700 hospitals [ 8 ] and 10 200 practicing hematologists and oncologists that could potentially see a patient [ 25 ]. In an attempt to capture this broader population, the alternative surveillance acquired hemophilia study collected bleed data from 99 patient cases submitted by 92 centers between 2008 and 2011 [ 26 ]. Ultimately, the demographics of those captured in HTRS are consistent with those of other acquired hemophilia data sets, suggesting these data are representative of the acquired hemophilia population in the United States.…”

Section: Discussionmentioning

confidence: 99%

Assessment of acquired hemophilia patient demographics in the United States

Kessler

Alice

Al‐Mondhiry

et al. 2016

Blood Coagulation &Amp; Fibrinolysis

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The Hemostasis and Thrombosis Research Society (HTRS) Registry was used to monitor the postapproval use of recombinant factor VIIa. The objective of this manuscript is to provide key insights on the demographics of patients with acquired hemophilia in the HTRS Registry. Acquired hemophilia patient registration in HTRS captured age; sex; comorbidities and predisposing conditions; first bleeding location; laboratory parameters; exposure to blood products, factor, and bypassing agents; and initiation of immune suppression/tolerance therapy. Overall, 166 patients with acquired hemophilia were registered in HTRS (83 women, 73 men, median age 70 years); the majority were non-Hispanic whites (61.4%). The most common comorbidities were autoimmune disease (28.4%) and malignancy (14.5%). The most common first site of bleeding was subcutaneous (27.1%); this was more common in whites (29.1%) than blacks (12.5%) and in non-Hispanics (26.4%) than Hispanics (11.8%). Blood product exposure was reported for 33.1% of patients; the most commonly reported product was packed red blood cells (28%). Of the 57 patients with outcome data available for immune tolerance therapy, 26 patients (46%) reported successful treatment, 13 reported unsuccessful treatment (23%), and 18 (32%) were receiving active treatment at the time of registration. The HTRS Registry final analysis provides the only current comprehensive look at acquired hemophilia in the US population, including details on underlying autoimmune diseases and malignancies. Pertinent to recognition and diagnosis of the disease, subcutaneous bleeding as a presenting bleeding symptom was more common in white and non-Hispanic individuals.

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Section: Discussionmentioning

confidence: 99%

Assessment of acquired hemophilia patient demographics in the United States

Kessler

Alice

Al‐Mondhiry

et al. 2016

Blood Coagulation &Amp; Fibrinolysis

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“…Two of the studies included > 150 patients [ 7 , 18 ], while three studies recruited 10 to 30 patients [ 23 , 25 , 26 ]. The number of bleeds was not reported in five of the 12 included studies [ 2 , 23 , 25 , 27 , 28 ]. Two studies did not report initial dose of rFVIIa [ 2 , 28 ] and were not included in the subsequently planned meta-analysis.…”

Section: Resultsmentioning

confidence: 99%

“…The number of bleeds was not reported in five of the 12 included studies [ 2 , 23 , 25 , 27 , 28 ]. Two studies did not report initial dose of rFVIIa [ 2 , 28 ] and were not included in the subsequently planned meta-analysis. The Zhang et al study that evaluated a very low dose (25–55 μg/kg) of rFVIIa was also excluded from the subsequently planned meta-analysis [ 29 ].…”

Section: Resultsmentioning

confidence: 99%

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Lessons from a systematic literature review of the effectiveness of recombinant factor VIIa in acquired haemophilia

Tiede

Worster

2018

Ann Hematol

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To conduct a systematic review of the literature reporting efficacy and safety of recombinant factor VIIa (rFVIIa) for the treatment of bleeding in acquired haemophilia and, if data permitted, undertake a meta-analysis of the current evidence. MEDLINE®, Embase®, and the Cochrane Central Register of Controlled Trials (CENTRAL) databases were searched for all studies on rFVIIa treatment in acquired haemophilia. Heterogeneity of included studies was measured using the inconsistency index (I2). Of the 2353 publications screened, 290 potentially relevant references were identified: 12 studies published in 32 publications met inclusion criteria. In total, 1244 patients and 1714 bleeds were included (671 patients received rFVIIa treatment for 1063 bleeds). In seven of 12 studies, the initial dose of Recombinant FVIIa was 90 ± 10 μg/kg. Recombinant FVIIa was used as first-line therapy in the majority of cases. Median number of doses administered ranged from 10 to 28. Between 68 and 74% of bleeds were spontaneous, whereas 4–50% were traumatic. Thirty-nine to 90% of bleeds were severe. Haemostatic effectiveness was > 90% in 5/6 studies for both patient and bleed level. Recombinant FVIIa had a favourable safety profile with low risk of general adverse events and thromboembolic-associated events. The heterogeneity of the studies and data precluded a meta-analysis. Recombinant FVIIa demonstrated effectiveness for the treatment of bleeds and had a good safety profile. It is apparent from these data that there is a need for more standardised measures of clinical effectiveness in acquired haemophilia to enable comparison and pooling of results in the future.Electronic supplementary materialThe online version of this article (10.1007/s00277-018-3372-z) contains supplementary material, which is available to authorized users.

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“…These data from SACHA support the findings from EACH2, confirming the efficacy and safety of bypassing agents in controlling bleeding in patients with AHA. Moreover, recent data from the Acquired Hemophilia Surveillance project have reaffirmed that rFVIIa is efficacious and well tolerated in AHA, with a low rate of thrombotic complications .…”

Section: Clinical Management: Treatment Of Acute Bleeding Episodesmentioning

confidence: 96%

Acquired haemophilia: an overview for clinical practice

Kessler

Knöbl

2015

European J of Haematology

115

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Acquired haemophilia is a potentially life-threatening bleeding disorder caused by the development of autoantibodies against coagulation factors, most commonly against factor (F) VIII (acquired haemophilia A; AHA). In around half of patients, an underlying disorder is associated with AHA; the remaining cases are idiopathic. Typically, the disorder presents with bleeding, ranging from mild to life- and limb-threatening, in patients with no personal or family bleeding history. Diagnosis involves an isolated prolongation of the activated partial thromboplastin time, without correction in mixing studies, low FVIII activity levels and evidence of a FVIII inhibitor. As AHA is rare, a lack of familiarity of the condition may result in delayed diagnosis, and prompt haemostatic control is required to reduce morbidity and mortality. Bypassing agents (recombinant activated factor VII or activated prothrombin complex concentrates) can be used to control acute bleeding, and immunosuppression is necessary to eradicate the inhibitor. As clinical trials in this rare and heterogeneous disease are difficult, current evidence comes from observational studies, including registries. This review will focus on the diagnostic and therapeutic challenges of AHA and summarise how understanding of this complex condition has increased based on recent registry data.

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A novel supplemental approach to capturing post-marketing safety information on recombinant factor VIIa in acquired hemophilia: the Acquired Hemophilia Surveillance project

Cited by 6 publications

References 2 publications

Assessment of acquired hemophilia patient demographics in the United States

Assessment of acquired hemophilia patient demographics in the United States

Lessons from a systematic literature review of the effectiveness of recombinant factor VIIa in acquired haemophilia

Acquired haemophilia: an overview for clinical practice

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