9 research outputs found
VWF/FVIII concentrates in high-risk immunotolerance : the resist studies
No full textBackground: Inhibitor eradication with immune tolerance induction (ITI) is the best long-term therapeutic strategy in Haemophilia A (HA) patients with inhibitors. Several parameters are known to be related to the ITI success. One that has more recently been proposed is the type of FVIII concentrate. Recent clinical findings indicate that plasma-derived FVIII products containing von Willebrand factor (VWF/FVIII) could have an impact on ITI success rate, even in poor prognosis patients.
Study design: Two key prospective clinical studies have been designed to confirm the previous results supporting the use of VWF/FVIII in ITI: RESIST experienced (RESISTexp) and RESIST naïve (RESIST-naïve).
RESIST: Exp is a prospective, nonrandomized study designed to assess rescue treatment with VWF/FVIII at high dosage (200 IU/kg daily) in patients who failed a previous ITI attempt with any dose of a VWF-free FVIII concentrate (plasma-derived or recombinant). RESISTnaïve is a prospective, controlled, randomized, open-label study comparing two types of FVIII concentrates (VWF-free FVIII and VWF/FVIII) in their ability to induce tolerance in high responding HA patients, with no previous ITI attempt and with poor prognosis for success. Both types of concentrates will be administered at high dosage (200 IU/kg daily). Enrolment criteria are: severe haemophilia A (FVIII 5 BU), any inhibitor level at study enrolment, and at least one of the following risk factors for ITI failure: (a) peak inhibitor titer > 200 BU, (b) titer at ITI start > 10 BU, (c) age > 7 years, (d) time between inhibitor occurrence and ITI > 2 years. Patient undergoing concomitant immunosuppressive treatment are not eligible for either study. Primary end point is success in achieving ITI defined as: complete or partial. Secondary endpoints are: ITI maintenance, time to success, safety/compliance to treatment and cost of care.
Conclusions: The results of RESIST studies will be crucial in understanding the role of VWF/FVIII in ITI outcome and will contribute to providing effective treatment for the devastating complication of FVIII antibody development in HA patient
Reversible skeletal changes after treatment with bevacizumab in a child with cutaneovisceral angiomatosis with thrombocytopenia syndrome
No full textDevelopment of computational biomechanical tools to assess the performance of the resurfaced hip joint
No full text482 Safety and Efficacy of Velaglucerase Alfa in Gaucher Disease Type 1 Patients Previously Treated with Imiglucerase
No full textResponse to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia
No full textSafety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase
No full textVelaglucerase alfa is a glucocerebrosidase produced by gene activation technology in a human fibroblast cell line (HT-1080), and it is indicated as an enzyme replacement therapy (ERT) for the treatment of Gaucher disease type 1 (GD1). This multicenter, open-label, 12-month study examined the safety and efficacy of velaglucerase alfa in patients with GD1 previously receiving imiglucerase. Eligible patients, ≥2 years old and clinically stable on imiglucerase therapy, were switched to velaglucerase alfa at a dose equal to their prior imiglucerase dose. Infusion durations were 1 hr every other week. Forty patients received velaglucerase alfa (18 male, 22 female; four previously splenectomized; age range 9-71 years). Velaglucerase alfa was generally well tolerated with most adverse events (AEs) of mild or moderate severity. The three most frequently reported AEs were headache (12 of 40 patients), arthralgia (9 of 40 patients), and nasopharyngitis (8 of 40 patients). No patients developed antibodies to velaglucerase alfa. There was one serious AE considered treatment-related: a grade 2 anaphylactoid reaction within 30 min of the first infusion. The patient withdrew; this was the only AE-related withdrawal. Hemoglobin concentrations, platelet counts, and spleen and liver volumes remained stable through 12 months. In conclusion, adult and pediatric patients with GD1, previously treated with imiglucerase, successfully transitioned to velaglucerase alfa, which was generally well tolerated and demonstrated efficacy over 12 months' treatment consistent with that observed in the velaglucerase alfa phase 3 clinical trial program
Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase
No full textEvaluating the impact of Attention Process Training (APT) on attention deficit in the early stages of recovery from stroke
No full textAttention deficits are a prominent sequel of stroke and impact negatively on rehabilitation outcomes. However, rehabilitation efforts are almost entirely concerned with the remediation of physical impairments that result from the stroke despite the involvement of attention in physical functioning. Attention Process Training (APT) is a cognitive retraining programme originally designed for the remediation of attention deficit following traumatic brain injury. However, the efficacy of APT post-stroke is not yet known, as to date, few studies have been conducted with small sample sizes. This study evaluated the effectiveness of APT in improving attention in stroke survivors within the 5 to 8 week period post-stroke. Seventy eight patients admitted to hospital with first-ever-stroke were identified as having an attention deficit by obtaining a score of 1 standard deviation below the normative mean on any of the following widely-used neuropsychological measures of attention; the Auditory Attention Quotient (AAQ) or Visual Attention Quotient (VAQ) of the Integrated Visual and Auditory Continuous Performance Test, (IVA-CPT), either trial of the Trail Making Test (TMT), the Paced Auditory Serial Addition Test (PASAT), or by 3 or more errors made on the left or right side of the Bells Cancellation Test. These measures were re-administered on completion of treatment. Participants were randomised to either the experimental group who received standard care and up to 30 hours of APT or to a control group that received standard care only. The primary outcome measure was the Full Scale Attention Quotient (FSAQ) of the IVA-CPT which is a measure of attention derived from both auditory and visual attention quotients. The secondary outcome measure was a health-related quality of life measure, the SF-36, (Short-Form-36). Both measures were administered before treatment and again on the completion of treatment. The results showed that on the primary outcome, the APT group showed improvement from baseline to post-treatment whereas the SC group had not. Significant improvement by the APT group was also demonstrated on two other measures of the IVA-CPT including the Auditory Attention Quotient and the Full Scale Response Quotient (a measure of impulsivity). On the quality of life measure neither the APT group nor the SC group demonstrated a significant change in scores. The results of this study provide further support for the efficacy of cognitive rehabilitation and in particular that APT is an effective cognitive treatment option for the remediation of attention deficit in the early stages of stroke recovery. The characteristics of stroke survivors who might benefit most from APT are identified as well as those factors that possibly influence the subjective experience of this particular intervention. The appropriateness of some measures such as the PASAT, the TMT, cancellation tests as well as continuous performance tests that are often used in research of attention deficit, are also discussed in the context of a stroke population. It is hopeful that the optimistic outcomes of this study will encourage further needed research in this area in order to inform stroke rehabilitation specialists to incorporate cognitive rehabilitation into predominantly physically-focussed programmes
