Stem Cell Transplantation in Patients Affected by Shwachman-Diamond Syndrome: Expert Consensus and Recommendations From the EBMT Severe Aplastic Anaemia Working Party.

Shwachman-Diamond syndrome is a rare disorder that can develop malignant and nonmalignant hematological complications. Overall, 10% to 20% of Shwachman-Diamond patients need hematopoietic stem cell transplantation (HSCT), but most centers have a limited experience and different approaches. The European Society for Blood and Marrow Transplantation-Severe Aplastic Anaemia Working Party promoted an expert consensus to propose recommendations regarding key issues in the management of Shwachman-Diamond patients with hematological complications.

Myasthenia gravis and paroxysmal nocturnal hemoglobinuria after thymectomy: A rare association.

Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare clonal autoimmune disease manifesting with hemolysis, thrombosis, or bone marrow failure. We present an atypical association of myasthenia gravis, aplastic anemia, and PNH occurring years after thymectomy. While this association might be extremely rare, it may not be coincidental as there is a common pathophysiology between PNH and aplastic anemia, with the latter reported in several thymoma/thymectomy cases.

Reporting of patient safety incidents in minimally invasive thoracic surgery: a national registered thoracic surgeons experience for improvement of patient safety.

Objectives: The reporting of patient safety incidents (PSIs) occurring in minimally invasive thoracic surgery (MITS) is crucial. However, previous reports focused mainly on catastrophic events whereas minor events are often underreported.

Methods: All voluntary reports of MITS-related PSIs were retrospectively extracted from the French REX database for 'in-depth analysis'.

Paroxysmal nocturnal hemoglobinuria and vascular liver disease: Eculizumab therapy decreases mortality and thrombotic complications.

A total of 2%-10% of patients with vascular liver disease (VLD) have paroxysmal nocturnal hemoglobinuria (PNH). Eculizumab reduces complement-mediated haemolytic activity in PNH. This study was aimed at assessing the impact of eculizumab on VLD outcome.

Development of a target product profile for a point-of-care cardiometabolic device.

Introduction: Multi-parameter diagnostic devices can simplify cardiometabolic disease diagnosis. However, existing devices may not be suitable for use in low-resource settings, where the burden of non-communicable diseases is high. Here we describe the development of a target product profile (TPP) for a point-of-care multi-parameter device for detection of biomarkers for cardiovascular disease and metabolic disorders, including diabetes, in primary care settings in low- and middle-income countries (LMICs).