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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare X-linked genetic disorder. On the contrary to its name, it is a multisystemic disease and various symptoms other than hemoglobinuria could be occurred. It could be life threatening especially because of thromboembolic events. In the last decade, a terminal complement inhibition with eculizumab approved with promising results for PNH patients. We conducted this study to evaluate the long term experience of eculizumab therapy from Turkey for the first time. Our cohort included 138 patients with PNH treated with eculizumab between January 2008 and December 2018 at 28 centers in Turkey. Laboratory and clinical findings at the time of diagnosis and after eculizumab therapy were recorded retrospectively. The median age was 39 (range 18-84) years and median granulocyte PNH clone size was 74% (range 3.06-99.84%) at the time of diagnosis. PNH with bone marrow failure syndrome was detected in 49 patients and the rest of 89 patients had classical PNH. Overall 45 patients (32.6%) had a history of any prior thrombotic event before eculizumab therapy and only 2 thrombotic events were reported during the study period. Most common symptoms are fatigue (75.3%), hemoglobinuria (18.1%), abdominal pain (15.2%) and dysphagia (7.9%). Although PNH is commonly related with coombs negativity, we detected coombs positivity in 2.17% of patients. Seven months after the therapy, increased hemoglobin level was seen and remarkably improvement of lactate dehydrogenase level during the treatment was occurred. In addition to previous studies, our real life data support that eculizumab is well tolerated with no serious adverse events and improves the PNH related findings., Competing Interests: None., (AJBR Copyright © 2021.)

PNH Education and Study Group (PESG) have been established in December 2013 as a non-profit, independent, medical organization www.pesg.org. Paroxysmal Nocturnal Hemoglobinuria (PNH) is a multi-systemic disease that should be treated with a multidisciplinary approach. Patients may apply to the clinics other than the hematology due to variability and diversity of clinical findings which lower the rate of diagnosis due to low awareness about PNH. PNH might be overlooked and diagnosis might be delayed. Regarding these, PESG was established with the collaboration of Immunology, Cardiology, Thorax Diseases (Pulmonology), Neurology, Gastroenterology, General Surgery and Urology specialists in addition to hematologists dealing with PNH. The PESG study group aims to increase the awareness about PNH, including training activities about PNH, strengthening the relations between clinics and planning of clinical studies as a goal. It is the first professional organization focusing on PNH, in Turkey.In this guideline, we want to facilitate the diagnosis attributes of physicians from all specializations that deal with PNH and its systemic complications. One can perceive this as a tailor made guideline of international guidelines but not a compilation.

We aimed to provide real-life information about the effectivity of different types of primary antifungal prophylaxis (AFP) in patients with acute myeloid leukemia (AML). Records of AML patients who received remission-induction chemotherapy between June 2010 and February 2013 were retrospectively reviewed. A total of 85 AML remission-induction chemotherapy cycles were identified in 80 patients. Fluconazole prophylaxis (FP) was administered in 29 cycles, and posaconazole prophylaxis was given in 56 cycles. Failure in the AFP was observed in 45 (57.9 %) out of 85 cycles. Any type of invasive fungal diseases were detected in 15 (26.8 %) out of 56 cycles receiving posaconazole and 15 (51.7 %) out of 29 cycles receiving fluconazole (p = 0.023). Relapsing or refractory AML, longer duration of neutropenia and FP were more common in patients with AFP failure. Multivariate logistic regression analysis showed that type of AFP (odds ratio (OR) 3.63; 95 % confidence interval (CI) 1.19-11.07), presence of neutropenia longer than 21 days (OR 3.96; 95 % CI 1.36-11.46), and refractory or relapsing AML (OR 6.09; 95 % CI 2.09-17.73) were independent factors associated with failure of AFP. We observed superiority of posaconazole on fluconazole in the prophylaxis of AML patients receiving remission-induction chemotherapy.