Liver fibrosis and its response to avapritinib in 2 patients with systemic mastocytosis

[No Abstract Available

Relationship Between Severity of Renal Amyloid Deposition and Clinical Outcomes in Non-AA Amyloidosis

Objective: Renal involvement is a common manifestation of systemic amyloidosis. Amyloid load can be predicted by histopathological grading of amyloid deposits in renal biopsy specimens. This study aimed to determine the relationship of renal amyloid deposition grade with clinical manifestations and outcomes in patients with biopsy-proven renal non-AA amyloidosis. Methods: This retrospective cohort study included 74 subjects with renal non-AA amyloidosis (52 light chain amyloidosis and 22 unclassified amyloidosis). Baseline characteristics and follow-up data were recorded. Pattern and quantity of amyloid deposition in glomeruli, interstitium, vessels, and tubulointerstitial changes were scored. Renal Amyloid Prognostic Score was obtained by addition of all scores and divided into 3 grades (Renal Amyloid Prognostic Score grades I, II, III). Results: In light chain amyloidosis group, the median follow-up was 11 (4-45) months. The baseline estimated glomerular filtration rate was significantly lower among patients with Renal Amyloid Prognostic Score grade III. Death-censored Renal survival was significantly lower among patients with Renal Amyloid Prognostic Score grade III. Renal Amyloid Prognostic Score grade III was a significant predictor of lower renal survival. Patient survival was not significantly different according to Renal Amyloid Prognostic Score grade. Receiving autologous stem cell transplantation treatment was associated with better patient survival. The type of amyloid could not be determined in 22 patients. In this group, baseline estimated glomerular filtration rate was significantly lower in patients with Renal Amyloid Prognostic Score grade III. Conclusions: In patients with light chain amyloidosis, baseline renal function is associated with Renal Amyloid Prognostic Score grade. Renal survival is significantly lower in patients with the highest Renal Amyloid Prognostic Score grade. However, patient survival is not significantly different according to Renal Amyloid Prognostic Score grade

Evaluation of Patients with PNH Treated By Eculizumab: Real World Data from Turkey

61st Annual Meeting and Exposition of the American-Society-of-Hematology (ASH) -- DEC 07-10, 2019 -- Orlando, FL[Abstract Not Available]Amer Soc HematolWOS:00057716460421

Evolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in turkey: a multicenter retrospective analysis

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.WOS:000672609100010PubMed: 3432229

Allogeneic hematopoietic stem cell transplantation in adult aplastic anaemia patients: Promising treatment modality

###EgeUn###European Soc Blood & Marrow Transplanta

Survival outcomes of unrelated donor source: A small retrospective study

###EgeUn###European Soc Blood & Marrow Transplanta

A new scoring system to predict survival in elderly advanced stage Hodgkin lymphoma patients

Predictive prognostic scoring (PS) systems are not primarily applicable to elderly patients with classical Hodgkin lymphoma (cHL). The objective of this study was to develop a PS system for these patients. The derivation cohort (DC) was utilized for model development, consisting of 97 variables. The resulting algorithm was named as Hodgkin’s Lymphoma Early Death in the Elderly within 12 months (HEDEL12). Internal and external validation cohorts (IVC and EVC) were employed for validation. A total of 286 patients were evaluated retrospectively. In DC 38 of 178 patients died within the first 12 months and overall survival (OS) at 12-month was 78.6%. Independent predictors of HEDEL12 were female sex, low albumin levels (<3.5 g/dL), and ECOG scores 2-4. According to HEDEL12 scores 0–1, OS at 12- months were 89.8% and 91.0% for IVC and EVC, respectively. The HEDEL12 scoring is useful in predicting the survival of advanced-stage cHL patients