The article is concerned with incidence, clinical features, response to therapy, and prognosis of patients with hypocellular myelodysplastic syndromes. Bone marrow (BM) cellularity <30% (or <20% in patients >70 yr) was found in 24 of 236 (10.2%) trephine biopsies. Median age was 61 yr, with significant male predominance (M/F=3.0) At diagnosis, median hemoglobin was 83 g/L, median platelet and neutrofil counts were 31x109/L and 1.2x109/L, respectively. According to FAB classification, 17 patients had RA, 6 had RAEB, and only 1 had RAEB-t. Beside marrow hypoplasia, the most prominent PH finding was megakaryocyte hypoplasia and dysplasia, found in two-thirds of cases, each. Comparison between hypocellular and normo/hypercellular MDS cases regarding clinicopathological features showed younger age, more severe cytopenia, less blood and BM blast infiltration, MK hypoproliferation, and more pronounced stromal reactions in former cases. Karyotypic abnormalities were present in 12.5% hypocellular cases, in contrast to 44.6% normo/hypercellular cases (p=0.0025). Eleven patients were treated with supportive therapy alone, six with danazol or androgens, six with immunosuppressive therapy, and one with LDARAC. However, complete or partial response was achieved in only four patients treated with danazol or androgens. None of the patients developed leukemia. Eleven patients died, so marrow insufficiency was the main cause of death. Median survival was 33 mo for hypocellular MDS, and 19 mo for normo/hypercellular MDS (p=0.09). The results confirm the existence of hypocellular variant of MDS, which seems to have better prognosis than those patients with normo/hypercellular disease.

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Use of intravenous immunoglobulins (IVIGs) is a well-established therapy in patients with acute or chronic autoimmune thrombocytopenic purpura (ITP). The aim of the present open, prospective study was to investigate efficacy and safety of the novel IVIG preparation BT681 (Biotest Pharma GmbH, Dreieich, Germany) in adult patients with chronic ITP (cITP). In order to fulfil high standards of purity and viral safety, an additional HPLC step has been introduced and validated virus removal and inactivating procedures are included in the manufacturing process of BT681. A total of 24 patients with platelet counts between 3/nl and 27/nl received a total dose of 2 g/kg body weight BT681 in standard dosage regimens: 1.0 g/kg/day for 2 days in 15 patients and 0.4 g/kg/day for five consecutive days in nine patients. Platelet response (platelet count > or =50/nl) and regression of haemorrhages were documented during the 28 days study period and safety parameters including adverse events, laboratory investigations and vital signs were regularly monitored. The overall response rate was 91.7%, that is 22/24 patients responded to BT681 treatment. The median time to platelet response was 3.0 days, the duration of response 25.5 days; the median max. platelet count 222/nl and the time to max. platelet count 7.5 days. During the entire observation period, platelet counts below baseline were only observed in five responders; in half of the patients, platelet counts were even above 50/nl at the end of follow-up. Corresponding to the good platelet response, the clinical symptoms improved markedly in the majority of patients within the first week after start of BT681 infusions with 88.9% overall regression of haemorrhages in comparison with baseline. The evaluation of platelet parameters showed a slight tendency in favour of the 5 days group, evaluation of haemorrhage severity slight advantages for the 2 days group. The adverse event data indicate that the therapy with BT681 was safe and well tolerated in both treatment groups even if minor effects were more frequent in patients treated with the 2 days regimen. The large majority of adverse events were known intolerabilities during the treatment phase described as mild and transient. The results demonstrate that the new immunoglobulin preparation BT681 is effective and safe in the treatment of adult patients with cITP.

In a retrospective study of 236 patients with primary myelodysplastic syndromes (MDS), 130 cases (55.1%) revealed myelofibrosis in bone marrow biopsies. It was observed that fibrosis mostly occurs focally or patchy, and collagen deposits were found very rarely (only four patients). The histopathology of bone marrow biopsies revealed several differences between fibrotic and non-fibrotic MDS: cellularity is significantly higher, dysmegakaryopoiesis is more pronounced, plasmocytes and mast cells are more often increased, and disturbance of marrow topography (particularly of the MK- and G-line) can be found more frequently in MDS with myelofibrosis. Reticulin fibrosis occurred in all subtypes of MDS; however, there was a higher incidence in chronic myelomonocytic leukemia. The frequency of abnormal growth of GM-progenitors was significantly higher in the MDS cases with myelofibrosis, compared to the cases without fibrosis. Clinical data showed significantly higher WBC, more frequent presence of immature granulocytes, and higher percentage of myeloblasts in peripheral blood and bone marrow in MDS with myelofibrosis compared to cases without myelofibrosis. Life expectancy was reduced to 13 mo, compared with 35 mo in MDS without fibrosis (p=0.00055). Time to leukemic transformation was 32 mo in MDS with fibrosis, compared with >56 mo in MDS without fibrosis (p=0.015). Myelofibrosis therefore seems to herald a poor prognosis.

Background: The role of natural killer (NK) cells in plasma cell diseases has not yet been fully characterized. Case Report: We present the case of a 47-year-old man with an extremely aggressive extramedullary plasmacytoma of the lung with associated cutaneous lesions, whose disease was accompanied by a significantly decreased number of NK cells (CD56+, CD16+, CD3–) in the peripheral blood, very low NK cell activity levels, and a decreased interleukin-2 production from cultured cells in vitro. Histology and immunohistochemistry of the lung and cutaneous lesions identified that the tumor was composed of clonal plasma cells which were CD38+++, CD138+++, lambda chain+, kappa chain–, and cytokeratin–. Bone marrow histology and cytology were initially normal. The disease progressed rapidly despite local radiotherapy and systemic chemotherapy, and the patient died shortly after diagnosis. Conclusions: Cutaneous involvement in extramedullary plasmacytoma represents a clinically aggressive variant of plasma cell tumor, which runs a rapid course and has associated devastating effects on the patient’s innate immune system.