Background Patients with homozygous familial hypercholesterolemia (HoFH) respond inadequately to existing drugs. We conducted a phase 3 study to assess the efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in adults with HoFH. Methods Twenty-nine subjects enrolled into a single-arm, open-label study and maintained current lipid lowering therapy from six weeks before baseline through at least week 26. Lomitapide dose was escalated based on safety and tolerability from 5 mg to a maximum of 60 mg/day. The primary endpoint was mean percent change from baseline in LDL-C at week 26, after which patients remained on lomitapide through week 78 for safety assessment. Findings Twenty-three subjects completed weeks 26 and 78. The median dose of lomitapide was 40 mg/day. LDL-C was reduced by 50% from baseline at week 26 (4·3 ± 2·5 mmol/L vs. 8·7 ± 2·9 mmol/L, p<0.0001). Eight subjects achieved LDL-C <2·6 mmol/L at this time point. LDL-C was reduced by 44% at week 56 and 38% at week 78 (p<0.0001 for both). Gastrointestinal symptoms were the most common adverse event. Four patients had aminotransaminase > 5× ULN that resolved after dose reduction or temporary interruption of lomitapide. No subject permanently discontinued treatment due to liver abnormalities. Liver fat content assessed by nuclear magnetic resonance spectroscopy (NMRS; n=20) was 1·0 ± 1·3 % at baseline, 8·6 ± 8·1% at week 26 and remained stable up to week 78 (8·3± 5·3%). Interpretation These data demonstrate that lomitapide had a robust and durable efficacy in lowering LDL-C in patients with HoFH with an acceptable safety and tolerability profile.

In this trial, ABVD therapy for 6 to 8 months was as effective as 12 months of MOPP alternating with ABVD, and both were superior to MOPP alone in the treatment of advanced Hodgkin's disease. ABVD was less myelotoxic than MOPP or ABVD alternating with MOPP.

Vectors based on human adenovirus (Ad) and adenovirus (NAB). Approximately two of three patients demonassociated virus (AAV) are being evaluated for human strated CD4 + T cells that proliferated to Ad antigens of gene therapy. The response of the host to the vector, in which most were of the TH 1 subset, based on cytokine terms of antigen-specific immunity, will play a substantial secretion. A substantially different pattern of immune role in clinical outcome. We have surveyed cohorts of norresponses was observed to AAV2. Although virtually all mal subjects and cystic fibrosis patients for pre-existing patients had Ig to AAV2, most of these antibodies were immunity to these viruses, caused by naturally acquired not neutralizing (32% NAB) and only 5% of patients had infections. A number of humoral and cellular assays to peripheral blood lymphocytes that proliferated in response adenovirus serotype 5 (Ad5) and adeno-associated virus to AAV2 antigens. These studies demonstrate marked hetserotype 2 (AAV2) were performed from serum and peripherogeneity in pre-existing immunity to Ad5 and AAV2 in eral blood mononuclear cells. Virtually all subjects had Ig human populations. The impact of these findings on outto Ad5 although only 55% of these antibodies neutralized come following gene therapy will require further study.

In patients with Stage III non-small-cell lung cancer, induction chemotherapy with cisplatin and vinblastine before radiation significantly improves median survival (by about four months) and doubles the number of long-term survivors, as compared with radiation therapy alone. Since three quarters of the patients still die within three years, however, further improvements in systemic and local therapy are needed.