ADAMTS13 levels in a plasma-derived FVIII concentrate: A potential therapeutic option for patients with congenital thrombotic thrombocytopenic purpura

Mori, Filippo; Nardini, Ilaria; Nannizzi, Sara; Crea, Roberto; Mathew, Prasad; Ziliotto, Nicole; Gringeri, A.

doi:10.1016/j.tru.2022.100120

Cited by 1 publication

(5 citation statements)

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“…The concept of lifelong ADAMTS13 replacement, and when to start it, also warrants further investigation. Additionally, rADAMTS13 may soon be available if approved; while this approach will likely achieve greater plasma ADAMTS13 activity than FVIII/VWF Koate , 16,25 comparative studies will be needed to evaluate relative clinical effectiveness.…”

Section: Discussionmentioning

confidence: 99%

“…These studies found that FVIII/VWF Koate contained substantially higher ADAMTS13 content (activity up to 9.08 U /100 IU FVIII and antigen level up to 8.42 U/100 IU FVIII in reconstituted concentrate) than other plasma‐derived concentrates 18,19 . Subsequent analyses further support the suitability of this product as a potential treatment for hTTP 16,20 …”

Section: Introductionmentioning

confidence: 87%

“…It is reconstituted and administered intravenously at home by the patient or a caregiver and stored at room temperature for up to 6 months or refrigerated until the expiration date 10,15 . The VWF (RCo):FVIII (activity) ratio of FVIII/VWF Koate is 1.4:1 16 …”

Section: Introductionmentioning

confidence: 99%

“…18,19 Subsequent analyses further support the suitability of this product as a potential treatment for hTTP. 16,20 A retrospective chart review reported data on eight patients with hTTP in the US being managed with FVIII/VWF Koate in lieu of FFP as on-demand therapy to treat recurring TTP episodes (microangiopathic haemolytic anaemia with thrombocytopenia and concomitant organ damage; two patients) or prophylactically to prevent TTP episodes (six patients). 10 All eight patients responded very well to FVIII/VWF Koate over a period ranging from 1 to 10 years, with a substantial reduction in the frequency of hTTP episodes and no reported allergic reactions or adverse events (AEs).…”

Section: Introductionmentioning

confidence: 99%

“…10,15 The VWF (RCo):FVIII (activity) ratio of FVIII/VWF Koate is 1.4:1. 16 Following publication of a single case report describing the successful use of FVIII/VWF Koate in the management of hTTP, 17 two independent laboratories quantified the ADAMTS13 content in several FVIII/VWF concentrates available in the US. 18,19 These studies found that FVIII/VWF Koate contained substantially higher ADAMTS13 content (activity up to 9.08 U /100 IU FVIII and antigen level up to 8.42 U/100 IU FVIII in reconstituted concentrate) than other plasmaderived concentrates.…”

Section: Introductionmentioning

confidence: 99%

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Long‐term follow‐up of patients with congenital thrombotic thrombocytopenia purpura receiving a plasma‐derived factor VIII (Koate) that contains ADAMTS13

Chrisentery‐Singleton,

Boggio,

Carcao

et al. 2023

Haemophilia

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BackgroundHereditary thrombotic thrombocytopenia purpura (hTTP) is an ultra‐rare disorder resulting from an inherited deficiency of ADAMTS13, a von Willebrand factor (VWF)‐cleaving metalloprotease. The plasma‐derived factor VIII/VWF Koate (FVIII/VWFKoate) has been shown to contain ADAMTS13, allowing for its use to treat hTTP at home by the patient/caregiver.AimBased on prior demonstration of safe and effective use of FVIII/VWFKoate in eight patients with hTTP, we conducted a retrospective study to gather additional data regarding the use of FVIII/VWFKoate for hTTP.MethodsThis was a multicentre, retrospective, noninterventional chart review of patients who had received FVIII/VWFKoate for the management of hTTP. Data collected included demographics, medical history, relevant family history, past use and tolerability of fresh frozen plasma, and details regarding FVIII/VWFKoate therapy.ResultsThe cohort included 11 patients (seven males, four females) with hTTP, ranging in age at study entry from 2 to 28 years. The average duration of FVIII/VWFKoate therapy was 4.8 years (range, 0.5–6.5 years). Among nine patients using FVIII/VWFKoate as prophylaxis, the normalized annual rate of breakthrough TTP episodes ranged from 0.2 to 1.1 episodes/year. All nine patients who received FVIII/VWFKoate prophylaxis had thrombocytopenia recorded at baseline, while eight (88.9%) did not have thrombocytopenia after using FVIII/VWFKoate. There was one AE (unspecified) attributed to FVIII/VWFKoate.ConclusionThese data suggest that FVIII/VWFKoate is a safe and well‐tolerated source of the missing ADAMTS13 enzyme in patients with hTTP, producing a marked reduction in thrombocytopenia prevalence, low frequency of TTP episodes, and with the added benefit of self‐ or caregiver‐administration.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 87%