Primary intravenous chemotherapy for group D retinoblastoma: a 13-year retrospective analysis.

BACKGROUND: Eye salvage rate for group D retinoblastoma using intravenous chemotherapy (IVC) as a primary modality is <50%. To report on 13 years' experience with the use of primary IVC for group D retinoblastoma. METHODS: A retrospective analysis of 64 group D eyes (52 patients) treated with primary IVC, from 2002 to 2014. RESULTS: The median age at presentation was 11.0 months (mean: 18.6, range: 0.6-144.0), 35 (67%) patients had bilateral disease, 38 (73%) germline disease and 8 (15%) cases were familial. In addition to IVC, patients received a median number of three treatments (mean: 6, range: 0-24), including thermotherapy/cryotherapy, plaque radiotherapy, intra-ophthalmic artery chemotherapy (IAC) and/or intravitreous chemotherapy. External beam radiotherapy (EBRT) was used in five eyes, all of which were eventually enucleated. In a median follow-up time of 55 months (mean: 64, range: 14-156), 63% of eyes were salvaged. By the Kaplan-Meier survival analysis, globe salvage rate was 83%, 70%, 59% and 45% at 1, 3, 5 and 10 years, respectively. There were no cases of metastatic spread from intraocular retinoblastoma and no deaths. IVC-related adverse events included febrile neutropenia in 21 (40%) patients and anaphylactic reaction to carboplatin in 2 (4%), all conservatively resolved. Of the patients receiving IAC, third and sixth nerve palsies were documented in two (10%) and one (5%) eyes, respectively. CONCLUSIONS: Primary IVC for group D eyes, with adjuvant treatments as required, was found to be a safe and efficient approach, achieving 63% eye salvage rate, no metastatic spread from intraocular retinoblastoma and no deaths. IAC has now replaced EBRT as a successful salvage treatment.

Glatiramer acetate and the glatiramoid class of immunomodulator drugs in multiple sclerosis: an update.

IMPORTANCE OF THE FIELD: MS is a chronic progressive inflammatory and neurodegenerative disease associated with autoimmune dysregulation. Glatiramer acetate (GA), a complex polypeptides mixture and first member of the glatiramoid class, is a first-line therapy for relapsing MS. New glatiramoids are under development. AREAS COVERED IN THIS REVIEW: Studies from a PubMed search with terms 'glatiramer' and 'glatiramoid' were evaluated, focussing on studies conducted between 2007 and 2010. WHAT THE READER WILL GAIN: We review newly discovered GA effects on innate and acquired immunity and results of recent clinical studies. GA delays conversion from a clinically isolated syndrome to definite MS and has clinical benefits comparable to those of IFN-beta drugs, but is more cost-effective and improves quality of life. Preclinical studies of protiramer, a higher molecular mass glatiramoid, showed unexpected toxicity in animals, resulting in discontinuation of drug development. TAKE HOME MESSAGES: GA is a cost-effective, safe and efficacious MS treatment with pleiotropic immunomodulation activity, is best prescribed early and may safely enhance outcomes when used with other immunomodulators. Protiramer experience indicates the potential for unexpected toxicity associated with new glatiramoids. The safety, efficacy and immunogenicity of new glatiramoids must be evaluated thoroughly.