None of the 24 patients in the pilot study suffered any serious ill effects, but in the new study several patients developed sudden, severe and unrelenting liver failure.
While most gene therapy trials have not been associated with the development of cancer, a notable exception was the treatment of X-linked Severe Combined Immuno-Deficiency (SCID-X1), where several patients developed a T-cell leukemia associated with vector integration near the proto-oncogenes LMO2, BMI1 and CCND2 (Hacein-Bey-Abina et al., 2003, 2008).
Clinical trials of a vaccine against Alzheimer disease came to an abrupt halt this year when several patients developed inflammation in the brain.
There have been no safety issues with any of the drugs, Dr. Vlasuk said, with the possible exception of a multiple myeloma trial, using very high doses of resveratrol, in which several patients developed a symptom common with the disease.
Several patients developed febrile neutropenia, nausea, or vomiting.
The majority of cases developed within a few days following vaccination and more often after the initial vaccination than subsequent vaccinations, although several patients developed recurrent inflammation with each vaccination.
However, in our comprehensive histopathological analysis, some findings were recurrent: most patients had both inflammatory and fibrotic components; several patients developed cirrhosis; individuals from three different families (A, C, and E) had histological findings consistent with hepatic nodular regeneration (Table 1).
Several patients developed late infections complicating their clinical course.
Since several patients developed more than one initial manifestation, we counted each manifestation separately.
Several patients developed asymptomatic elevations in amylase ∼1 week after the start of treatment.
