MORO, Isabel et al. ISSN Summary Acquired Von Willebrand disease is an unusual situation arising within the context of self-immune diseases, lymphoproliferative and mieloproliferative disorders. Clinical symptoms may be very proteiform, affecting several systems, and in this case it presented complications resulting from tumor adsorption: acquired Von Willebrand disease. Chemotherapy with thalidomide, cyclophosphamide and dexamethasone was indicated for the base disease and there was favourable evolution, the hemorrhagic syndrome remitted and there was a tendency to crasis normalization, the same as globular and platelets values ongoing normalization after six series of treatment.
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Print Download. Examples: Cancer AND drug name. Pneumonia AND sponsor name. How to search [pdf]. For these items you should use the filters and not add them to your search terms in the text field. Print Download Summary. Review by the Competent Authority or Ethics Committee in the country concerned. As of 1. EU Clinical Trials Register. Search tools. Select Date Range: to. Select Rare Disease:.
IMP with orphan designation in the indication. Orphan Designation Number:. Results Status: Trials with results Trials without results. Clear advanced search filters.
Date on which this record was first entered in the EudraCT database:. Title of the trial for lay people, in easily understood, i. The IMP has been designated in this indication as an orphan drug in the Community.
Committee on Advanced therapies CAT has issued a classification for this product. Combination product that includes a device, but does not involve an Advanced Therapy. Severe von Willebrand disease. Evaluate the efficacy of the product as a treatment for bleeds. Evaluate the efficacy of the product as prophylaxis for surgeries.
Evaluar la eficacia del producto como tratamiento en hemorragias. Evaluate the clinical safety, as well as the immunogenicity and thrombogenicity of the product. Evaluate the tolerance to the product administration.
Patients less than 6 years of age 3. Patients that do not adequately respond to treatment with desmopressin 4. Signed informed consent by the patient's legal representative mother, father o tutor. Pacientes que no respondan adecuadamente al tratamiento con desmopresina 4. Firma del consentimiento informado por parte del representante legal del paciente madre, padre o tutor.
The subject has been diagnosed of acquired VWD2. Patients bleeding at the time of the first infusion or the 10 days prior to the infusion 3. Subjects treated with desmopressin or another VWF containing FVIII concentrate during the 5 days prior to the infusion of the investigational product 4. The subject is known to have history of anaphylactic reaction s to blood or blood components 7.
Subjects diagnosed with metabolic diseases that are not clinically controlled, such as diabetes mellitus, that could potentially interfere with the interpretations of the study The subject is participating in another clinical study involving an investigational treatment, or participated within the past month The subject is unlikely to adhere to the protocol requirements of the study Pacientes que presenten enfermedad de von Willebrand adquirida.
Pacientes con historia conocida de reacciones graves o frecuentes a productos derivados del plasma. Sospecha de condiciones que pudieran afectar al cumplimiento del protocolo por parte del paciente. Pharmacokinetic parameters will be assessed within a maximum of 15 days after the recruitment visit. Whenever a bleeding treated in the hospital occurs throughout the 12 month follow-up period.
Whenever a surgery or invasive procedure occurs throughout the 12 month follow-up period. Siempre que ocurra un sangrado tratado en el hospital a lo largo de los 12 meses de seguimiento. Evaluate product's clinical security, inmunogenicity and trombogenicity detecting presence of FVIII and FVW inhibitors and evaluating clinical trombosis in patients undergoing quirurgic or invasive procedures. Evaluate the product's administration tolerance detecting adverese reactions, including clinically significant changes in vital signs or lab paramenters.
The clinical safety and tolerance to Fanhdi will be assessed since recruitment and throughout the 12 month follow-up period. Immunogenicity will be measured before the first infusion of the investigational product, every three months throughout the follow-up period and when there exists a clinical suspicion of the formation of inhibitors. Thrombogenicity will be measured in all subjects undergoing surgery or an invasive procedure. The trial involves single site in the Member State concerned.
Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial. Pediatric patients under 6 years of age. Plans for treatment or care after the subject has ended the participation in the trial if it is different from the expected normal treatment of that condition.
2012, Number 04
Print Download. Examples: Cancer AND drug name. Pneumonia AND sponsor name. How to search [pdf]. For these items you should use the filters and not add them to your search terms in the text field.
Síndrome de Heyde
Only comments seeking to improve the quality and accuracy of information on the Orphanet website are accepted. For all other comments, please send your remarks via contact us. Only comments written in English can be processed. A rare bleeding disorder marked by the same biological anomalies as those seen in hereditary von Willebrand disease VWD but which occurs in association with another underlying pathology, generally in elderly patients without any personal or family history of bleeding anomalies. Prevalence is unknown, but Acquired von Willebrand syndrome AVWS is a rare disease that is underdiagnosed, with just over cases reported in the literature so far. The bleeding manifestations are similar to those occurring in hereditary VWD prolonged bleeding after trauma, epistaxis, ecchymoses and gastrointestinal bleeding associated with angiodysplasia.