Onyx Pharmaceuticals, Inc., an Amgen subsidiary.

Srdeční amyloidóza je charakteristická klinicky významnou extracelulární depozicí amyloidu v srdečních tkáních, která bývá obvykle doprovázena i postižením dalších orgánů v závislosti na konkrétním typu amyloidového proteinu. Srdeční postižení je nejdůležitějším prognostickým faktorem přežívání nemocných, a to především s AL amyloidózou. Časná diagnostika srdeční amyloidózy je tak zcela zásadní pro volbu dalšího terapeutického postupu. Tento přehledový článek si klade za cíl informovat podrobně o patogenezi, projevech a metodách diagnostiky srdeční amyloidózy. Zároveň uvádí přehled současných terapeutických možností, které mají velký potenciál zlepšit dosud poměrně špatnou prognózu postižených jedinců.

Intravenous immunoglobulin (IVIg) has an established role in the treatment of immune thrombocytopenia (ITP). The safety and efficacy of a new ready-to-use IVIg 10% formulation (octagam(®) 10%) were investigated in a prospective phase III study in 116 adult patients with ITP (platelet count ≤20×10(9)/l). Sixty-six patients had chronic ITP and 49 were newly diagnosed. Patients received octagam 10% 1 g/kg/day on two consecutive days; infusion rate was adjusted according to tolerability to a maximum of 0·12 ml/kg/minute. Eighty per cent of patients attained the primary efficacy endpoint of clinical response (platelet count ≥50×10(9)/l within 6 days of dosing). The median time to response was 2 days and the median duration of response was 12 days; mean response duration was 24·1 days. octagam 10% was well tolerated and effective in this population representative of adult patients with ITP, even at the maximum infusion rate of 0·12 ml/kg/minute, without unexpected safety issues.

Abstract. The primary objective of the present prospective study was to compare the diagnostic performance of conventional radiography (CR) and whole-body low-dose computed tomography (WBLDCT) with a comparable radiation dose reconstructed using hybrid iterative reconstruction technique, in terms of the detection of bone lesions, skeletal fractures, vertebral compressions and extraskeletal findings. The secondary objective was to evaluate lesion attenuation in relation to its size. A total of 74 patients underwent same-day skeletal survey by CR and WBLDCT. In CR and WBLDCT, two readers assessed the number of osteolytic lesions at each region and stage according to the International Myeloma Working Group (IMWG) criteria. A single reader additionally assessed extraskeletal findings and their significance, the number of vertebral compressions and bone fractures. The radiation exposure was 2.7±0.9 mSv for WBLDCT and 2.5±0.9 mSv for CR (P= 0.054). CR detected bone involvement in 127 out of 486 regions (26%; P<0.0001), confirmed by WBLDCT. CR underestimated the disease stage in 16% and overestimated it in 8% of the patients (P=0.0077). WBLDCT detected more rib fractures compared with CR (188 vs. 47; P<0.0001), vertebral compressions (93 vs. 67; P= 0.010) and extraskeletal findings (194 vs. 52; P<0.0001). There was no correlation observed between lesion size (≥5 mm) and its attenuation (r=-0.006; P=0.93). The inter-observer agreement for the presence of osteolytic lesions was κ= 0.76 for WBLDCT, and κ= 0.55 for CR. The present study concluded that WBLDCT with hybrid iterative reconstruction technique demonstrates superiority to CR with an identical radiation dose in the detection of bone lesions, skeletal fractures, vertebral compressions and extraskeletal findings, which results in up-or downstaging in 24% patients according to the IMWG criteria. The attenuation of osteolytic lesions can be measured with the avoidance of the partial volume effect.