Clinical use of Haemate(R) P in inherited von Willebrand's disease: a cohort study on 100 Italian patients

Federici, Augusto B.; Castaman, Giancarlo; Franchini, Massimo; Morfini, Massimo; Zanon, Ezio; Coppola, Antonio; Tagliaferri, Annarita; Boeri, E; Mazzucconi, Maria Gabriella; Rossetti, Gina; Mannucci, Pier Mannuccio

doi:10.3324/haematol.11124

Cited by 71 publications

(78 citation statements)

References 18 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…One additional source of long bleeding times among apparently healthy individuals is von Willebrand's disease, which occurs in about 10% of the Italian population. 19 Even though at birth, von Willebrand factor multimers are physiologically at high levels, 20 we are uncertain whether any of our neonates with long bleeding times might be on the basis of von Willebrand's disease or other platelet dysfunction syndromes. It would have been useful to see whether the neonates with the highest bleeding times on day 1 still had high values on days 10 and 30.…”

Section: Discussionmentioning

confidence: 99%

Template bleeding times of 240 neonates born at 24 to 41 weeks gestation

et al. 2008

View full text Add to dashboard Cite

Objective: The template bleeding time is a test used to assess the hemostatic effectiveness of platelet/endothelial interactions. A modified template bleeding time, described over 15 years ago by Andrew et al., has been used to test this aspect of hemostasis in term and preterm infants, but questions remain regarding the effect of gestational age and postnatal age on results. The effect of the platelet count (platelets per microliter blood) and the circulating platelet mass (nl platelets per microliter blood) on the bleeding time of neonatal intensive care unit (NICU) patients also require better definition.Study Design: We measured template bleeding times on 240 neonates at Ospedale A Perrino, in Brindisi, Italy; studying groups of n ¼ 60 at the following gestational ages; <28 weeks, 29 to 32 weeks, 33 to 37 weeks and X38 weeks. In each group of 60 neonates, 20 were studied on the first day after birth, 20 were studied on day 10 and 20 were studied on day 30. A multivariate analysis was performed to examine various associations with bleeding time.Result: Bleeding times, on the first day of life, were shorter as gestational age increased. Those born <33 weeks gestation had bleeding times about twice that of those X38 weeks (P<0.001). Bleeding times tended to shorten between days 1 and 10. Little or no further shortening occurred between days 10 and 30, and by day 30 they were not statistically different between the various gestational age groups. No independent effect on bleeding time could be ascribed to gender, platelet count or circulating platelet mass, but independent effects were found for hematocrit (P<0.02) and gestational age (P<0.001). Conclusion:On the first day of life, preterm neonates have a longer bleeding time than do term neonates. By day of life 10, the bleeding times at all gestational ages are shorter and are indistinguishable on the basis of gestational age at birth. Additionally, since platelet counts as low as 110 000 per microliter did not prolong the bleeding time, we see no benefit of administering a platelet transfusion to a stable, nonbleeding, NICU patient with mild thrombocytopenia (platelet count 100 000 to 150 000 per microliter).

show abstract

Section: Discussionmentioning

confidence: 99%

Template bleeding times of 240 neonates born at 24 to 41 weeks gestation

et al. 2008

View full text Add to dashboard Cite

show abstract

“…Total ED to Haemate ® P up to the first study visit varied among patients, with a tendency to more limited exposure in patients with VWD type 1 compared with the other disease types (> 70% of type 1 patients with ED 1-24 days). During the last 12 months before entering the study, 42 patients of the entire cohort (34.7%) underwent surgery, dental extractions and invasive procedures, with a clinical response scored as excellent or good in 95% of cases [9]. In the same period, the majority of patients (75.2%) had either no bleeding episodes or <5 episodes requiring treatment with VWF/FVIII concentrates.…”

Section: Characteristics Of the Study Populationmentioning

confidence: 99%

“…At each follow-up visit, the clinical response to Haemate ® P was assessed by the clinician as recommended by the European Medicines Agency Guideline on the clinical investigation of human plasma-derived VWF products and rated as previously reported [9,11]. The response was rated as excellent when it was clinically not different from normal on surgery and invasive procedures, or when optimal and fast control of spontaneous bleeding was achieved; good, when mildly abnormal, partial or delayed control of spontaneous bleeding, or slight transient oozing from surgical wounds; moderate, when moderately abnormal haemostasis bleeding not fully controlled but no need for additional therapy; poor, when no improvement at all with continuation of bleeding and need for additional or alternative therapies [9]. The safety of the treatment was also monitored and suspected adverse events related to the treatment were recorded in a standardized case report form.…”

Section: Variables Recordedmentioning

confidence: 99%

“…The concentrate (infusion of 4 9 10 3 IU per event, median; 12 9 10 3 IU per patient, median) was given 60 min (median) before the procedure and after surgery (timing of postoperative treatment was dependent on surgery type and patient bleeding tendency) [ Table 4]. These patients received a total of 234 postoperative, surgery-related infusions (median 7.0 per patient, range [1][2][3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19]. The median number of postoperative infusions required to treat one event was five (range 1-16).…”

Section: Characteristics Of the Study Populationmentioning

confidence: 99%

“…Patients with VWD of either gender and of any age were eligible if they had been already treated with Haemate ® P. Patients were diagnosed according to the criteria of the Scientific Standardization Committee on VWF of the International Society of Thrombosis and Haemostasis, and VWD types were determined as previously reported [1,9].…”

Section: Study Populationmentioning

confidence: 99%

See 2 more Smart Citations

Efficacy and safety during formulation switch of a pasteurized VWF/FVIII concentrate: results from an Italian prospective observational study in patients with von Willebrand disease

Castaman

Coppola

Zanon³

et al. 2012

Haemophilia

Self Cite

View full text Add to dashboard Cite

Summary. Von Willebrand disease (VWD) is an inherited bleeding disorder caused by the quantitative or qualitative deficiency of von Willebrand factor (VWF). Replacement therapy with plasma-derived VWF/factor VIII (FVIII) concentrates is required in patients unresponsive to desmopressin. To assess the efficacy, safety and ease of use of a new, volume-reduced (VR) formulation of VWF/FVIII concentrate Haemate ® P in patients requiring treatment for bleeding or prophylaxis for recurrent bleeding or for invasive procedures. Pharmacoeconomic variables were also recorded. Data were analysed using descriptive statistics. This was a multicentre, prospective, observational study. Consecutively enrolled patients received Haemate ® P VR according to their needs, and were followed for 24 months. Of the 121 patients enrolled, 25.6% had type 3 VWD and more than 40% had severe disease. All patients were followed for 2 years, for a total of 521 visits. Ondemand treatment was given to 61.9% of patients, secondary long-term prophylaxis to 25.6% and prophylaxis for surgery, dental or invasive procedures to 45.5%. The response to treatment was rated as good to excellent in >93-99% of interventions. The new formulation was well tolerated by all patients with no report of drug-related adverse events. The switch to volume-reduced Haemate ® P was easy to perform and infusion duration was decreased twofold compared with the previous formulation. Volume-reduced Haemate ® P was at least as effective and well-tolerated as the previous formulation.

show abstract