AbstractIntroductionMeasuring health‐related quality of life (HRQoL) in haemophilia patients provides a comprehensive patient's functional and the impact of the disease and its treatment.AimThis study aimed to assess the HRQoL of haemophilia children and related factors.MethodsWe conducted a cross‐sectional study from 6 April 2019 to 29 April 2019. We recruited children aged 8–16 years old who were members of the Yogyakarta Province Branch, Indonesian Haemophilia Society. Children filled in the Indonesian Haemo‐QoL short‐version questionnaire that consists of nine dimensions, and the score ranges from 0 to 100, in which a higher score shows a higher impartment.ResultsForty‐four children participated in this study. The mean age was 11.99 + 2.49. The median age of the first bleeding episode was 5 years, and the median frequency of spontaneous bleeding during the previous year was 13.5. All children received antihemophilic factor (AHF) on‐demand; most (81.8%) received AHF in the advanced symptoms. The mean total score of HRQoL was 42.7 ± 15.5, with the highest impairment in the family dimension, with a mean score of 68.0 ± 22.3. The severity of haemophilia was significantly correlated with the total score of HRQoL and dimensions of physical health, feeling, view, and family (effect size ranges from 0.30 to 0.42). The frequency of bleeding was significantly related to the total score of HRQoL and the feeling dimension (effect size of 0.47 and 0.34).ConclusionThe HRQoL of our study was lower than in countries where AHF prophylactic is provided. More severe haemophilia and more frequent bleeding significantly lower HRQoL.

2023-11-01·Haemophilia

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

Article

作者: Steele, MacGregor ; Torres, Marcela ; Boggio, Lisa N ; Carcao, Manuel D ; Khan, Osman ; Rodino, Frank J ; Chrisentery-Singleton, Tammuella ; Rajasekhar, Anita ; Ibrahimi, Sami ; Carpenter, Shannon L ; Mahajerin, Arash ; Sharma, Vivek

AbstractBackgroundHereditary 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.

2022-01-01·Haemophilia

Low‐dose immune tolerance induction therapy in children of Arab descent with severe haemophilia A, high inhibitor titres and poor prognostic factors for immune tolerance induction treatment success

Article

作者: El‐Ekiaby, Magdy ; Hassab, Hoda ; Elalfy, Mohsen ; Elalfy, Omar ; Elghamry, Islam ; Andrawes, Nevine

AbstractIntroductionImmune Tolerance Induction (ITI) is the first‐choice therapy to eradicate Factor VIII (FVIII) neutralizing antibodies in patients with haemophilia A (HA). There is limited published data on ITI from East Mediterranean countries.AimTo assess the effectiveness of a low‐dose ITI regimen to eradicate FVIII neutralizing antibodies in children with severe HA and high‐titre inhibitors.MethodsA prospective, single‐arm study was conducted in children with HA (FVIII < 1 IU/dl), high‐titre inhibitors and poor prognostic factors for successful ITI. Patients were treated with ∼50 IU/kg plasma‐derived FVIII containing von Willebrand factor (pdFVIII/VWF) concentrate (Koate‐DVI, Grifols) three times a week. Time to achieve tolerance, total and partial success were analysed after ITI. Annual bleeding rate (ABR), number of target joints, FVIII recovery and school absence were compared before and after ITI.ResultsTwenty patients with median (range) age of 6.2 (3–12) years and pre‐ITI inhibitor titre of 36.5 (12–169) BU were enrolled. ITI lasted ≤12 months (early tolerization) in 45% of patients. Median follow‐up was 12 months (3–22) and total response rate was 80% (60% total success; 20% partial success). Patients with two and three poor prognosis factors achieved overall success rate of 60% and 50%, respectively. ABR, target joints and school absence were reduced after ITI by 60%, 50% and 44.1%, respectively. In successful ITI tolerized patients, FVIII recovery was 90 (60–100)%.ConclusionA low‐dose ITI therapy using a pdFVIII/VWF concentrate achieved at least partial tolerance in 80% of patients, and reduced annual bleeds in children with high inhibitor titres and at least one poor prognosis factor for ITI treatment success.

100 项与 Antihemophilic Factor (Human) (Grifols) 相关的药物交易

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适应症	国家/地区	公司	日期
获得性因子 VIII缺乏症	美国	Grifols Therapeutics, Inc.	1997-01-24

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ISTH2020	N/A	血友病A	13	Antihemophilic factor (human)	蓋鏇積構鬱願繭構蓋鬱(齋網積網選窪願憲鑰艱) = 壓鏇膚醖襯糧範淵衊構鬱壓齋顧網襯構襯襯構 (簾鏇衊衊壓齋網範齋築 )	-	2020-07-12