Background: Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency.
Methods: We treated 50 patients with ADA-SCID (30 in the United States and 20 in the United Kingdom) with an investigational gene therapy composed of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a self-inactivating lentiviral vector encoding human . Data from the two U.
Background: Strimvelis (autologous CD34+ cells transduced to express adenosine deaminase [ADA]) is the first ex vivo stem cell gene therapy approved by the European Medicines Agency (EMA), indicated as a single treatment for patients with ADA-severe combined immunodeficiency (ADA-SCID) who lack a suitable matched related bone marrow donor. Existing primary immunodeficiency registries are tailored to transplantation outcomes and do not capture the breadth of safety and efficacy endpoints required by the EMA for the long-term monitoring of gene therapies. Furthermore, for extended monitoring of Strimvelis, the young age of children treated, small patient numbers, and broad geographic distribution of patients all increase the risk of loss to follow-up before sufficient data have been collected.
View Article and Find Full Text PDFWe report the presentation of a 43-year-old female with an unusual acute complication from an inherited blood dyscrasia. After a provisional working diagnosis of pulmonary embolus, the patient was finally diagnosed with spontaneous haemorrhage from extramedullary haematopoietic foci within the thorax.
View Article and Find Full Text PDFOral Surg Oral Med Oral Pathol Oral Radiol Endod
March 2007
Objective: Temporomandibular disorder (TMD) and chronic daily headache (CDH) are chronic problems affecting the head and face. The aim of this study was to compare TMD and CDH, in terms of cognitive factors, including illness perceptions and coping strategies.
Study Design: A total of 102 patients, 18 to 70 years of age, classified into 2 groups, TMD or CDH, were recruited in this study from a variety of centres in London.