Objective:To evaluate the safety and efficacy of eltrombopag combined with immunosuppressive therapy in patients with aplastic anemia (AA) in China.Methods:We investigated and analyzed the clinical data of AA patients from 14 hematological treatment centers who were treated with oral eltrombopag for at least 3 mon.Results:We enrolled 56 AA patients, including 19 treatment-na?ve patients and 37 IST-refractory patients. The median administration period for eltrombopag was 7 (3-31) months, and the median maximum stable dosage was 75 mg/d (50-150 mg/d) . The 3-month hematological response (HR) rate was 60%, and the complete response (CR) rate was 30% in 10 SAA patients who were treated with first-line eltrombopag and standard IST (ATG+CsA) . Eight of 9 eltrombopag and CsA ± androgen first-line treated SAA patients responded (8/9, 89%) and 4 (44%) gave CR. The overall HR and CR rates were 79% and 52.6%, respectively, among these 19 patients by the end of the follow-up period. Of the 19 AA patients who were refractory to CsA ± androgen, 11 achieved HR (57.9%) at 3 mon, and the best HR rate was 44% in standard IST (ATG+CsA) refractory 18 patients after eltrombopag treatment. Fifty-one percent of the patients experienced mild or moderate adverse events, and gastrointestinal discomfort was the most common adverse effect reported by the study subjects.Conclusion:Adding Eltrombopag in first-line IST can accelerate the acquisition and improve the quality of hematological responses in AA patients. AA with relatively more residual hematopoietic cells may be well treated with eltrombopag and non-ATG IST. Eltrombopag can be used as salvage therapy for CsA±androgen refractory patients. Eltrombopag was generally safe and well tolerated by AA patients in China.

Objective To analyze the image characteristics of whole-body bone planar scan and SPECT/CT imaging in patients with polyostotic bone fibrous dysplasia ( PFD) . Methods Twenty-three pa-tients with PFD (12 males, 11 females, age: 10-77(31.4±3.4) years) between June 2007 and March 2017 were enrolled. Twenty-one patients were confirmed pathologically and 2 was diagnosed by follow-up re-sults. The images of 99Tcm-methylene diphosphonate (MDP) whole-body bone scan and SPECT/CT imaging were retrospectively analyzed. Results Bone involvement in the extremities was the most common and lesions in the lower and right limbs were more than those in the upper and left limbs. Lesions were frequently unilat-erally on whole-body bone planar images in 18 of the other 23 patients ( 78. 3％) . Among them, 15/16 with limb lesions showed no bone deformation in limbs, while the enlargement and deformity were more common in the skull, ribs and pelvis. Vertebral involvement was found in 7 of 23 patients (30.4％), and the hand and foot bone involvement was found in 6 of 23 patients (26.1％). Most lesions (96.9％, 248/256) presented high or mod-erate abnormal uptake, which distributed in a stripe shape in the extremities, ribs and skull. On SPECT/CT ima-ges, the ground glass, vegetable sponge and mixed lesions showed higher uptake compared with the cystic le-sions. Conclusions The PFD has certain characteristics on whole-body bone scan. SPECT/CT imaging can reflect pathological, blood flow and metabolic changes of PFD.

Objective: To investigate the serum expression and influencing factors of hepcidinin patients with classical paroxysmal nocturnal hemoglobinuria (PNH) . Methods: Retrospective analysis of 36 classical PNH patients from 2016.3 to 2017.3. Serum hepcidin concentration was measured by ELISA method. The relationship between serum hepcidin concentration and erythropoiesis and iron homeostasis parameters was evaluated. Results: The median serum hepcidin level of 36 classical PNH patients was 32.03 (23.11, 118.48) μg/L, it was significantly lower than of 181.42 (106.80, 250.53) μg/L in 292 normal control subjects (z=-5.107, P<0.001) . The median serum hepcidin of 56.41 (44.60, 95.06) μg/L in PNH patients with normal ferritin was significantly lower than that in normal controls. The median serum hepcidin concentration 23.75 (21.77, 30.35) μg/L in iron deficiency PNH patients was lower than that in the normal ferritin PNH patients. However, the median serum hepcidin level of classical PNH with elevated ferritin patients 336.19 (304.19, 375.08) μg/L was significantly higher not only than that of normal ferritin and iron deficiency PNH ones, but also than that of normal control subjects. Regression analysis showed that serum ferritin, transferrin saturation and serum albumin level were independent influencing factors of serum hepcidin level in patients with classical PNH. Conclusion The decreased serum hepcidin level in patients with classical PNH was mainly influenced by iron metabolism factors.

Objective: To reveal the genetic characteristics of erythrocyte membrane protein in hereditary spherocytosis (HS) in China. Methods: Next-generation sequencing technology was used to detect mutations in genes of erythrocyte membrane proteins in 51 clinically diagnosed HS patients. The relationship between gene mutations and clinical phenotypes was analyzed. Results: Mutations in erythrocyte membrane protein genes were detected in 37 patients, including 17 with ANK1 mutations (17/37, 45.9%), 14 with SPTB mutations (14/37, 37.8%), and 5 with SLC4A1 mutations (5/37, 13.5%). One patient carried both heterozygous ANK1 mutation and SPTB mutation (1/37, 2.7%). SPTA1 and EPB42 mutation was not fou nd in any patient. Nonsense mutations (36.8%) and missense mutations (31.6%) were most common. Of the 38 mutations detected, 34 were novel mutations and have not been reported elsewhere (89.5%). Sixteen HS patients underwent parental genetic validation, 6 patients (37.5%) inherited gene mutation from parents and 10 (62.5%) were de novo. The peripheral blood cell parameters of HS patients were not related to the mutant genes and gene mutation types. However, it seems that HS patients with mild clinical status are prone to carry SPTB mutations while more patients with severe clinical status have ANK1 mutations. Conclusions ANK1 and SPTB are the most common mutant genes in Chinese HS patients, mainly with missense mutations and nonsense mutations. There was no significant correlation between the mutation of HS related genes and the severity of HS.

Objective: To investigate the relationship between the eosin-5′-maleimide (EMA) binding test and the clinical severity of hereditary spherocytosis (HS). Methods: A total of 258 un-splenectomize HS patients were consecutively enrolled. Correlation of hemoglobin concentration, hemolytic parameters, compensating erythropoiesis and the EMA binding test were evaluated. Results: 258 (128 male and 130 female) patients were included in this study, including 91 compensatory hemolysis patients, 53 patients with mild anemia, 78 patients with moderate anemia and 36 patients with severe anemia. The median age at diagnosis was 23 (2-70) years. The median decreased fluorescence intensity of EMA binding test was 29.97% (16.09%-47.34%) and the average intensity was (29.70±6.28) % of 258 HS patients. The decreased EMA binding fluorescence intensity correlated with MCV (r=-0.343, P<0.001) and MCHC (r=0.223, P<0.001). There was no relationship between EMA fluorescence intensity and absolute reticulocyte count (r=0.080, P=0.198) , reticulocyte percentile (r=-0.015, P=0.813) , IBIL levels (r=-0.009, P=0.902) , HGB levels (r=-0.067, P=0.280). Evaluated as a quartile variable, EMA fluorescence intensity was not correlated with anemia severity (C=0.150, P=0.746). Conclusion EMA binding test does not related to anemia levels and has no major clinical implications for disease severity in HS.

BACKGROUND:Bone marrow mesenchymal stem cel transplantation is a hot spot in the treatment of ischemic cerebrovascular diseases, and CT brain perfusion scan is mostly used to observe the improvement in the blood flow in the ischemic region. However, X-ray irradiation during CT brain perfusion may affect the viability of transplanted bone marrow mesenchymal stem cel s, and then influence the therapeutic efficacy. OBJECTIVE:To investigate the effect of CT brain perfusion dose on the viability of bone marrow mesenchymal stem cel s. METHODS:Passage 5 bone marrow mesenchymal stem cel s from Sprague-Dawley rats were selected and randomized into three groups, with 6 tubes in each group. One of the six tubes in each group was randomly selected with no irradiation, and the remaining five tubes in each group were subjected to CT brain perfusion scans 1-5 times, respectively. After scanning, the cel number was counted after 10-day continuous culture and cel growth curve of each tube was drawn. Cel cycle was detected by flow cytometry, and cel viability was measured by MTT method. RESULTS AND CONCLUSION:After 10 days of continuous counting, the number of cel s per tube showed no difference (P>0.05), and cel growth curves were basical y coincided. Moreover, there was no significant difference in the cel cycle and cel viability (P>0.05). Experimental findings show that the CT brain perfusion scan has no effect on the viability of bone marrow mesenchymal stem cel s.

OBJECTIVETo investigate the sensitivity and specificity of eosin-5'-maleimide (EMA)assay for the diagnosis of hereditary spherocytosis (HS), and to verify the stability of reagent and samples.

METHODSEMA flow cytometry test, NaCl-osmotic fragility test and acidified glycerol lysis test were performed using peripheral blood samples from 80 patients with HS and 44 patients with other blood diseases, the sensitivity and specificity of the three methods were compared, and the feasibility of EMA binding test was estimated. The stability of EMA reagent and HS samples stored at different temperatures were tested.

RESULTSAmong the 124 tested samples, the sensitivity and specificity of EMA binding test was 0.925 and 0.954, that of NaCl-osmotic fragility test was 0.950 and 0.455, and that of acidified glycerol lysis test was 1.000 and 0.318, respectively. Although the sensitivity of NaCl-osmotic fragility test and acidified glycerol lysis test was a little higher than that of EMA binding test, the specificity of the former two methods was poor, they couldn't clearly distinguish whether spherocytosis is hereditary spherocytosis. The experiment results showed that EMA was sensitive to the temperature and should not be stored in a small aliquots at -80 ℃ over a period of 6 months. The stability of the HS sample was better, 6 days storage at 4 ℃ and 3 days storage at room temperature had no influence on the results.

CONCLUSIONEMA binding test by flow cytometry showed good sensitivity and specificity for HS diagnosis. EMA reagent should be stored at-80 ℃ and the HS samples should be tested within 6 days storage at 4 ℃ and 3 days at room temperature.

Ankyrins ; blood ; deficiency ; Eosine Yellowish-(YS) ; analogs & derivatives ; Flow Cytometry ; Hematologic Tests ; Humans ; Sensitivity and Specificity ; Spherocytosis, Hereditary ; blood ; diagnosis

OBJECTIVETo identify the characteristics of chronic natural killer cell lymphocytosis (CNKL).

METHODSThe clinical data of eight cases defined by the World Health Organization classification was retrospectively analyzed and related literatures were reviewed.

RESULTSHalf of the 8 patients were asymptomatic. Among them, the most common abnormalities were leukocytosis with the median of 11.8(4.5-20.0)×10⁹/L and high proportion of lymphocytes [0.78(0.51-0.89)], while anemia and thrombocytopenia only in 1 patient respectively. The absolute CD3⁻CD16⁺ NK cell count with the median of 5.7(2.4-9.6)×10⁹/L increased in peripheral blood. By the end of follow-up, except one case was lost, the other seven patients were in stable condition,including four cases without any medications and three patients receiving chlorambucil or glucocorticoid.

CONCLUSIONAs an indolent chronic lymphoproliferative disease, CNKL was presented with mild clinical symptoms and increased number of CD3⁻CD16⁺ NK cells in peripheral blood. Patients with symptoms could be treated with immunosuppressive therapy while those asymptomatic could be followed up without intervention.

Adult ; Aged ; Chronic Disease ; Female ; Follow-Up Studies ; Humans ; Killer Cells, Natural ; Lymphocytosis ; blood ; Male ; Middle Aged ; Prognosis

Objective To strengh the awareness of acute T-B cell biphenotypic leukemia.Methods Four new cases of acute T-B cell biphenotypic leukemia were reported and the related literature were reviewed.Results Fourteen patients with acute T-B cell biphenotypic leukemia.including 4 diagnosed at our hospital and other 10 cases reported in literature,were retrospectively analyzed.Similar clinical fleatures as the typical acute lymphocytic leukemia(ALL) were presented.Ten of 14 cases were male and were within 17 to 46 years old.The disease were refactory to the conventional ALL chemotherapy regimens and deteriorated progressively.Six patients died within 12 months after their diagnosed with the median survival time of ten months. Conclusion Acute T-B cell biphenotypic leukemia is one of the most rare type of leukemia.A better understanding of the clinical and hematological features of this type of leukemia and new therapeutic strategies are needed.

Objective To study the dietary nourishment of adult patients with leukemia and compare acute leukemic patients with chronic leukemic patients. Methods Adopting dietary review of 24 hours and seven consecutive days of dietary records method to obtain the food category and quantity of 122 patients with acute leukemia and 10 patients with chronic leukemia. Using statistic software SPSS11.0 to calculate the patients'intake of various kinds of nutfiments. and the difiences between acute and chronic leukemic patients were analyzed. Results The rate of most ontrients of patients'intake reaches RNI/AI is lower,especially vitamin A,vitamin C and caleium.There's a tendency that intake diet,energy and nourishments of acute leukemic patients is lower than that of those chronic leukemic patients. Conclusion There is a tendency of unbalanced dietary intakes in leukemic patients.including the low intakes.There is the tendency that nutritional status of acute leukemic patients iS poorer than that of chronic leukemic patients.