British journal of haematology
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This study compared the diagnostic value of Whole-Body Ultra Low-Dose computed tomography (WBULDCT) with that of Spinal Magnetic Resonance Imaging (SMRI) in identification of spinal bone marrow involvement in patients with Multiple Myeloma (MM). Thirty-five patients with histologically proven MM underwent WBULDCT and dedicated SMRI. Unenhanced WBULDCT was performed on a 256-slice scanner, with 120 kV and 40 mAs. ⋯ The concordance in spinal distribution of lesions was 61·6% on cervical, 71·5% on dorsal, 86·4% on lumbar and 94·4% on sacral, while for the pattern of disease, it was 56·1% for the focal and 88·7% for the combined pattern. Cohen's kappa index was 0·85 (P < 0·001) assessing an excellent agreement. WBULDCT represents a useful diagnostic tool in the detection of spinal involvement of MM patients, offering detailed information about extra-axial involvement, which could be potentially missed with dedicated SMRI.
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Eltrombopag is well established in treatment of severe immune thrombocytopenia (ITP) and is increasingly commonplace in second-line management. A role is also suggested for both bridging therapy for surgery, as well as treating thrombocytopenia due to non-immune aetiologies. We present the largest single-centre experience with eltrombopag, with our cohort of 62 patients. ⋯ We conclude that eltrombopag is both effective and well tolerated as therapy in severe ITP. It is also advantageous in ITP patients who do not normally require therapy, but need a temporary platelet count boost pre-procedure. Furthermore, there are potentially far wider implications for the use of eltrombopag in counteracting thrombocytopenia beyond ITP, which merit further investigation.
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Pulmonary hypertension (PH) is a rare, but life-threatening, adverse event in patients treated with tyrosine kinase inhibitors (TKIs), such as dasatinib, but has not been fully evaluated in patients treated with imatinib or nilotinib. We used echocardiography to noninvasively assess the incidence of PH in 105 patients with chronic myeloid leukaemia (CML) treated with imatinib (n = 37), nilotinib (n = 30) or dasatinib (n = 38). The mean triscupid regurgitation peak gradient (TRPG), which reflects pulmonary arterial pressure, was 22·7 mmHg in the imatinib group, 23·1 mmHg in the nilotinib group and 23·4 mmHg for dasatinib group. ⋯ In addition, there was a tendency toward correlation of TRPG value and age or TKI treatment duration. These results suggested that treatment with not only dasatinib, but also imatinib and nilotinib, can be associated with subclinical PH. Noninvasive echocardiography is useful for screening, especially in older patients with long-term TKI treatment.
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The inherited bone marrow failure syndromes (IBMFS) typically present with significant cytopenias in at least one haematopoietic cell lineage that may progress to pancytopenia, and are associated with increased risk of cancer. Although the clinical features of the IBMFS are often diagnostic, variable disease penetrance and expressivity may result in diagnostic dilemmas. ⋯ This has advanced understanding of the underlying biology of the IBMFS and been essential in improving clinical management and genetic counselling for affected patients. Herein we review the clinical features, underlying biology, and new genomic discoveries in the IBMFS, including Fanconi anaemia, dyskeratosis congenita, Diamond Blackfan anaemia, Shwachman Diamond syndrome and some disorders of the myeloid and megakaryocytic lineages.