Summary. In this report we analyse the presenting features of a series of patients diagnosed with Waldenstro Èm macroglobulinaemia (WM) in Spain over the last 10 years. Criteria for diagnosis required a serum monoclonal IgM protein > 30 g/l and . 20% bone marrow lymphocytes. Two hundred and seventeen patients were included in the study, with a median age of 69 years and male/female ratio of 2:1. The most common symptoms at diagnosis were anaemia (38%), hyperviscosity (31%), B symptoms (23%), bleeding (23%) and neurological symptoms (22%). Sixty-one patients (27%) were asymptomatic at diagnosis and, to date, 32 of them have not received chemotherapy. Variables predicting a shorter survival free of therapy were haemoglobin , 12´5 g/dl and high b 2 microglobulin (b2M). The 83% of patients who did receive treatment were distributed as follows: chlorambucil/prednisone (43%), intermittent chlorambucil (11%), continuous chlorambucil (26%), cyclophosphamide/vincristine/ prednisone (COP, 13´5%) and other (6´5%). Response to therapy was complete in 2%, partial in 48% and minor in 10%. Finally, 28% and 13% of patients presented stable and progressive disease, respectively, which was more common among patients treated with COP. Progression-free survival was 43% at 5 years, with three independent predictors for shorter progression-free survival (PFS): COP treatment, age . 65 and B symptoms at diagnosis. The 10-year projected overall survival (OS) was 55%. The two most frequent causes of death were development of second malignancies (31%), or infections (19%). The two main variables predicting a poor OS were hyperviscosity and high b2M. In summary, this study favours the use of chlorambucil-based therapy as the standard treatment for WM, and describes a subset of patients who should be considered as suffering a smouldering form and who therefore do not require treatment for a long period of time.

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Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by anti-ADAMTS13 antibodies. Caplacizumab is approved for adults with an acute episode of iTTP in conjunction with PEX and immunosuppression. The objective of this study is to analyze and compare the safety and efficacy of caplacizumab versus the standard of care and assess the effect of the concomitant use of rituximab. A retrospective study from the Spanish TTP Registry of patients treated with caplacizumab vs those who did not receive it was conducted. A total of 155 iTTP patients (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5% p<0.05) and less refractoriness (4.5% vs 14.1% p<0.05) than those that were not treated. Time to clinical response was shorter when caplacizumab was used as initial treatment vs caplacizumab used after refractoriness or exacerbation. The multivariate analysis showed that its use in the first 3 days after plasma exchange (PEX) was associated with a lower number of PEX (OR 7.5, CI 2.3-12.7; p<0.05) and days of hospitalization (OR 11.2, CI 5.6-16.9; p<0.001) compared to standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared to the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared to standard of care regimens. When administered within the first 3 days after PEX it also provided a faster clinical response, reducing hospitalization time and the need for PEX.

Background: Iron deficiency anemia (IDA) is highly prevalent in women of child-bearing age. However, their nonhematological symptoms have been overlooked. This study aims to analyze the nonhematological features and symptoms of IDA in a group of women of reproductive age and the changes occurred during iron therapy. Materials and Methods: IDA women underwent dietary, physical activity, menstrual blood loss, and cognitive function assessment at baseline. Hematological and biochemical parameters were analyzed. Executive attention was tested by the flanker task and working memory by the 2-back task. Oral iron therapy (ferrous sulfate) was given to 35 women for 8 weeks and the changes in iron status, biochemical markers, cognitive function, and nonhematological symptoms were evaluated. Results: Patients presented nonhematological symptoms: pica, 32.4%; cheilitis, 20.6%; restless legs syndrome (RLS), 20.6%; diffuse hair loss, 55.9%; and ungual alterations, 38.2%. Two or more symptoms were present in 58.8% of women. Serum iron and working memory were correlated at baseline. Multivariate analyses show associations (odds ratio [OR], 95% confidence interval [CI]) between pica and reaction time in the working memory test (OR 2.14, 95% CI 1.19-3.87, p = 0.012); RLS with total serum protein (OR 0.08, 95% CI 0.06-0.92, p = 0.043); and cheilitis with mean corpuscular hemoglobin (OR 0.388, 95% CI 0.189-0.799, p = 0.01). Pica, cheilitis, and RLS completely resolved with iron therapy, and ungual alterations and hair loss improved in 92.3% and 84.2% of women, respectively. Better performance in executive attention and working memory was observed after iron therapy. Conclusions: More attention should be given to the nonhematological manifestations of IDA to improve the quality of life of menstruating women.

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