Romiplostim for Primary Immune Thrombocytopenia in Routine Clinical Practice: Results from a Multicentre Observational Study in Germany.

INTRODUCTION: The effectiveness and safety of romiplostim were evaluated by immune thrombocytopenia (ITP) phase (newly diagnosed/persistent/chronic) at romiplostim initiation. METHODS: This is a post hoc analysis of a prospective, German, multicentre, observational study in adults with ITP who received ≥1 dose of romiplostim. Follow-up data were collected for ≤2 years. Outcomes included overall platelet response (≥1 platelet count ≥50 × 109/L at 2-24 weeks after romiplostim initiation) or durable platelet response (≥75% of measurements ≥50 × 109/L at 14-24 weeks) and adverse drug reactions (ADRs), evaluated by ITP phase. RESULTS: Data from 96 patients were analysed (newly diagnosed, n = 18; persistent, n = 25; chronic, n = 53). During the 2- to 24-week follow-up, overall platelet response was achieved in 100% (95% confidence interval: 81.5-100), 100% (86.3-100), and 96.2% (87.0-99.5) of patients with newly diagnosed, persistent, or chronic ITP, respectively, and platelet responses were durable in 88.2% (63.6-98.5), 65.0% (40.8-84.6), and 69.4% (54.6-81.7) of patients. During the 2-year follow-up, ADRs occurred in 24.0-35.8% of patients across phases. Two patients with chronic ITP experienced bone marrow ADRs; no thrombotic ADRs occurred. CONCLUSION: Romiplostim was effective and well tolerated in patients with newly diagnosed, persistent, or chronic ITP in routine clinical practice.

[Hemolytic anemias and vitamin B12 deficieny]. / Hämolytische Anämien und Vitamin-B12-Mangel.

Hemolytic anemias consist of corpuscular, immun-hemolytic and toxic hemolytic anemias. Within the group of corpuscular hemolytic anemias, except for the paroxysmal nocturnal hemoglobinuria (PNH), all symptoms are caused by underlying heredetiary disorders within the red blood cell membran (hereditary spherocytosis), deficiencies of red cell enzymes (G6PDH- and pyrovatkinase deficiency) or disorders in the hemoglobin molecule (thalassaemia and sickle cell disease). Immune-hemolytic anemias are acquired hemolytic anemias and hemolysis is caused by auto- or allo-antibodies which are directed against red blood cell antigens. They are classified as warm, cold, mixed type or drug-induced hemolytic anemia. Therapy consists of glucocorticoids and other immunsuppressive drugs. Pernicious anemia is the most important vitamin B12 deficiency disorder. Diagnosis relies on cobalamin deficiency and antibodies to intrinsic factor. The management should focus on a possibly life-long replacement treatment with cobalamin.

[Monoclonal gammopathy--multiple myeloma]. / Monoklonale Gammopathie und Multiples Myelom. Vom Zufallsbefund zur lebensbedrohlichen Erkrankung.

The diagnosis of monoclonal gammopathy of undetermined significance (MGUS), requires both the detection of monoclonal gammopathy by immunofixation on the one hand, and the exclusion of signs of multiple myeloma (MM) on the other. The risk of an MGUS evolving into an MM is about 1% per year. The possibility of an MM should be considered in particular in elderly patients with an elevated ESR, anemia, recurrent infections and hypercalcemia. The diagnosis of MM is established on the basis of a constellation of major and minor criteria that includes characteristic results of serum and urine electrophoresis and immunofixation, routine lab and bone marrow (plasma cell infiltration) studies as well as complications (anemia, renal failure, hypercalcemia, osteolysis). Standard chemotherapy comprises melphalan-prednisone or chemotherapy VAD. Improved results have been achieved with high-dose chemotherapy plus various forms of stem cell transplantation. To treat recurrences, thalidomide and bortezomib are now available.