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Objective To investigate the overall and subgroup efficacy, subgroup differences, predictors of efficacy and safety of roxadustat in the treatment of refractory non-severe aplastic anemia (NSAA) and low-risk myelodysplastic syndromes (LR-MDS). Methods Patients with refractory NSAA and LR-MDS who were admitted to the Department of Hematology, Peking Union Medical College Hospital from August 2020 to December 2022 were enrolled. All patients received first-line standard treatment, including recombinant human erythropoietin (rhEPO) for at least 3 months before roxadustat treatment. All patients received roxadustat 2.5 mg/kg every other day for at least 3 months, and were followed up for at least 8 months. The clinical characteristics of patients, roxadustat efficacy, predictors of efficacy, adverse effects,relapse and disease clonal evolution were analyzed. Results A total of 40 patients including 24 refractory NSAA and 16 LR-MDS were included. median age was 56 (18 - 81) years and 40% were males. 65% of the patients were transfusion dependent. Median follow-up was 21 (9 -34) months. 22.5%, 25.0%, 47.5%, 55.0%, 57.5%, 60.0% and 50.0% of the patients achieved haematological improvement-erythroid (HI-E) at months 1, 2, 3, 4, 5, 6, and end of the follow-up period, respectively, and no factors affecting HI-E were identified.The hemoglobin change from baseline was statistically different between the two groups at the end of the follow-up period. 50% of patients were relieved from transfusion dependence. Adverse reactions were reported in 22.5% of patients. 28.5% of patients relapsed after achieving HI-E, with a median time to relapse of 7 (4 - 12) months. No clonal evolution was observed at the end of the follow-up period. Conclusions Our preliminary findings suggested that Roxadustat may be effective for patients with NSAA or LR-MDS refractory to conventional therapies and rhEPO, with mild adverse effects and low relapse rate.The degree of hemoglobin improvement may be better in the refractory NSAA patients. [ABSTRACT FROM AUTHOR]

Background and Objectives: To explore the effect of nutrition management under ERAS concept in patients with spinal tuberculosis. Methods and Study Design: The study was conducted in an orthopedic ward of a tertiary grade A special hospital in Beijing. The patients admitted from January 1, 2021 to June 27, 2023 were screened for inclusion. The qualified patients were randomized into experimental group or control group. The experimental group received perioperative nutrition management under the concept of ERAS while the control group received routine perioperative management in hospital. The data was collected on the next day of admission, the next day and the sixth day after operation, including laboratory indicators (lymphocyte count, hemoglobin level, etc), intraoperative bleeding volume, postoperative exhaust, defecation time, drainage volume, albumin infusion amount, nutritional risk score, length of stay, hospitalization costs, etc. Univariate analysis and multivariate analysis correcting for gender, age, and baseline values were performed using SPSS24.0. Results: A total of 127 patients with spinal tuberculosis completed the study. Compared with the control group, the intraoperative blood loss (p=0.028) in the experimental group was significantly reduced, the postoperative exhaust time (p=0.012) and defecation time (p=0.012) were significantly shortened, and the nutritional status (p<0.001) was significantly improved. Besides, the results of multivariate analysis are robust after correcting potential confounding factors. Conclusions: Nutrition management under the concept of ERAS is helpful to reduce intraoperative bleeding, promote postoperative flatus and defecation, and improve nutritional status in patients with spinal tuberculosis, which may further improve their clinical outcome and prognosis. [ABSTRACT FROM AUTHOR]

The standard therapies for autoimmune cytopenias, including idiopathic thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA) and Evans syndrome (ES), are corticosteroids and intravenous immunoglobulin G. However, the recurrence rate is high. Data from 80 patients with ITP, AIHA and ES who were refractory to corticosteroids/relapsed and were treated with tacrolimus from January 2018 to January 2019 in Peking Union Medical Colleague Hospital were reviewed retrospectively. There were 24 males and 56 females, with a median age of 43 (14–81) years, including 66 with ITP, 11 with AIHA and 3 with ES. The median disease duration before tacrolimus was 16 (2–432) months. The complete response (CR) rates were 30.3%, 63.6% and 0%; the overall response (OR) rates were 63.6%, 72.7% and 66.7% for ITP, AIHA and ES, respectively; and the median time to response was 3 (2–10) months. In a median of 18 (10–24) months of follow-up time, 21.4% of ITP patients relapsed at a median time of 7 months. No relapse was found for patients with AIHA and ES. Side effects occurred in 16.3% of patients, including elevated creatinine (N = 3, 3.8%), gastrointestinal reactions (N = 3, 3.8%), and pulmonary infection (N = 2, 2.5%), and resulted in 3 patients stopping tacrolimus. The OR rate was found to be related with age (P = 0.01) but not with sex (P = 0.62), the duration of disease (P = 0.66), tacrolimus concentration (P = 0.99) or disease type (P = 0.84). Tacrolimus can achieve a durable response with mild side effects in patients with steroid-refractory/relapsed autoimmune cytopenias. Patients with younger age had a better response. [ABSTRACT FROM AUTHOR]

• AVA showed acceptable effects on patients with secondary AA after chemoradiotherapy. • AVA showed a dose-dependent effect on patients. • Previous ELT and age had no effect on patients' response to AVA. • Safety and effectiveness of AVA were further confirmed by this study. Aplastic anemia (AA) secondary to radiotherapy presents a difficult situation in the treatment of both the malignant tumor and AA itself. We aimed to evaluate the efficacy of avatrombopag (AVA), a thrombopoietin receptor agonist, in patients with AA secondary to chemoradiotherapy. In this retrospective study, patients with malignant tumors who were diagnosed with AA after radiotherapy and chemotherapy and accepted AVA between September 2020 and October 2021 at Peking Union Medical College Hospital were selected. A total of 34 patients were enrolled, including 13 (38.2%) men, with a median age of 60 (20–71) years. At a median of 8 (6–18) months of follow-up, the overall response rates (ORRs) at 1, 3, and 6 months were 32.4%, 55.9%, and 58.8%, respectively, and the complete response rates (CRRs) were 5.9%, 14.7%, and 23.5%, respectively. The median time to respond was 3 (1–6) months. In total, 15.0% of patients relapsed during follow-up, but no clonal evolution was noticed. Mild side effects were observed in 17.6% of patients without drug withdrawal. At the end of follow-up, 17.6% of patients had tumors relapsed. Four patients died, three from tumor relapse and one from cerebral hemorrhage. The ORR and CRR did not correlate with eltrombopag before AVA (p > 0.05) but increased when the total exposure of AVA increased (p = 0.011), and the threshold for AVA response was a cumulative dose > 3,000 mg (p = 0.013). AVA yielded good response and tolerance in patients treated for AA secondary to chemoradiotherapy, and a higher dose may correlate with better response. [ABSTRACT FROM AUTHOR]

With large patient population and complement inhibitors naïve background, the characteristics patients with paroxysmal nocturnal hemoglobinuria (PNH) in China have not been well studied, especially for different subtypes. We retrospectively reviewed patients with complete data who visited Peking Union Medical College Hospital (PUMCH) from 2009 to 2019 and had been followed up for more than 2 years. Five hundred and twelve patients were enrolled including 56.3% males and 43.7% females. The median age at disease onset was 33 (9∼80) years. Most were aged 21∼40 years (50.6%). 52.1%, 46.3% and 1.6% of the patients had classic PNH, bone marrow failure (BMF)/PNH and subclinical PNH, respectively. Symptoms of classic PNH were associated with hemolysis, whereas bleeding was more common in BMF/PNH patients. Classic PNH had higher survival rate, larger PNH clone size, higher lactate dehydrogenase (LDH) level and lower ferritin level than BMF/PNH. Although the rate of thrombosis was similar in the classic PNH and BMF/PNH (P = 0.66), those with BMF/PNH had higher chance of renal impairment (P < 0.05). Immunosuppressive agents was more common use in BMF/PNH (P < 0.05), but glucocorticoids, iron supplements and anticoagulants were more common used in classic PNH (P < 0.05) patients. Less evolution to myeloid malignancies was observed in classic PNH than in BMF/PNH (P = 0.02). The major causes of deaths were thrombosis (29.6%), hemorrhage (18.5%) and infections (18.5%). Patients with classic PNH and BMF/PNH have different clinical profiles, and we described a more hemolytic features of PNH in China which might be improved with complement inhibitors. [ABSTRACT FROM AUTHOR]

No agreement had been reached on the treatment of patients with pure red cell aplasia (PRCA) secondary to indolent malignancies. Data was collected from patients with acquired PRCA from May, 2014 to May, 2018 in Peking Union Medical College Hospital. Tumor-associated PRCA and primary PRCA patients were matched at a ratio of 1:2 with compatible baseline characteristics. All patients had been treated with CsA or sirolimus for at least 6 months with the efficacy and adverse events recorded. Twelve tumor-associated PRCA patients (3 thymoma, 8 lymphoproliferative disorders, and 1 smoldering multiple myeloma) with stable underling disease and 24 acquired primary PRCA patients were selected. 83.3% tumor-associated PRCA patients and 100% primary PRCA patients (P = 0.436) responded to immunosuppression therapy (IST) at a median of 2.5 and 3.5 months (P = 0.137), respectively. No different was found in side effects. The ORR at the end of a median of 21.5-month follow-up was 75% and 70.8% (P = 0.795), respectively. No tumor progression was reported except one secondary patient had lymphoma relapse after 2 years of IST and was given chemotherapy again. These results suggested IST had similar effect, safety on patients with tumor-associated, and primary PRCA patients when the tumors were stable. [ABSTRACT FROM AUTHOR]

Eltrombopag has shown a promising effect on patients with refractory/relapsed aplastic anemia (AA). However, data in Asian patients are limited due to the short launching time. Data from relapsed/refractory AA patients treated with eltrombopag in Peking Union Medical College Hospital from December 2017 to May 2019 were analyzed retrospectively. Totally 41 patients including 37 non-severe AA and 4 severe AA were analyzed with a median age of 47 (13~81) years old. The dosage of eltrombopag varies from 25 mg every other day (qod) to 100 mg every day (qd) (median 75 mg qd) with the duration of 13 (3~23) months. The overall response rates were 51.2%, 58.5%, and 55.2% at 3-month, 6-month, and 1-year follow-up. Seventy-five percent of patients achieved the best response below the dosage of 75 mg qd at 3 (1–6) months. Two patients stopped eltrombopag after achieving complete remission and remained stable. Two patients relapsed and evolved to myelodysplastic syndrome. Adverse events included gastrointestinal discomfort, hepatic toxicity, and skin reactions, which were mild and controllable. Eltrombopag is effective for Chinese relapsed/refractory AA patients with a relatively lower dose and acceptable side effects. [ABSTRACT FROM AUTHOR]

For patients with pure red cell aplasia (PRCA), cyclosporine (CsA) is the first line therapy. Occasionally, some patients who suffer from renal insufficiency cannot tolerate CsA. To explore the efficacy and tolerance of sirolimus treatment for those patients, twelve PRCA patients with renal insufficiency from May 2014 to May 2018 in Peking Union Medical College Hospital were enrolled, treated with sirolimus, and followed up at the median time of 16 (10-50) months. Eleven patients (91.7%) responded to sirolimus, with 58.3% complete response (CR) and 41.7% partial response (PR). The median time to achieve the optimum effect was 4 (1-7) months. The serum creatinine level remained stable or even reduced during the treatment period for eleven patients. Seven patients (58.3%) reported adverse events during sirolimus therapy, including increased blood glucose, infection, skin rash, elevated triglyceride or total cholesterol, and elevated serum creatinine compared with baseline. No treatment-related death was noticed during the follow-up time. Three patients relapsed with an overall response rate of 75.0% at 1 year. These results suggested that sirolimus was effective and tolerable for patients with PRCA complicated with renal insufficiency. [ABSTRACT FROM AUTHOR]

The adverse effects caused by PM2.5 have drawn extensive concern and it is of great significance to identify its spatial distribution. Satellite-derived aerosol optical depth (AOD) has been widely used for PM2.5 estimation. However, the coarse spatial resolution and the gaps caused by data deficiency impede its better application at the urban scale. Additionally, obtaining accurate results in unsampled spatial areas when PM2.5 ground sites are insufficient and distribute sparsely is also a challenging issue for PM2.5 spatial distribution estimation. This paper aimed to develop a model, i.e., spatially local extreme gradient boosting (SL-XGB), combining the powerful fitting ability of machine learning and optimal bandwidths of local models, to better estimate PM2.5 concentration at the urban scale by using Beijing as the study area. This paper adopted simplified high-resolution MODIS aerosol retrieval algorithm (SARA) AOD at 500 m resolution as the major independent variable, hence, ensuring the estimation can be operated at a fine scale. Moreover, the extreme gradient boosting (XGBoost) model was adopted to fill the gaps in SARA AOD, thus improving its availability. Then, based on full-covered SARA AOD and other multisource data, the SL-XGB model, integrating multiple local XGBoost models and particular optimal bandwidths, was trained to estimate PM2.5 concentration. For comparison, SL-XGB and two other models, XGBoost and geographically weighted regression (GWR), were evaluated by 10-fold cross validation (CV). The sample-based CV results reveal that the SL-XGB performed the best as assessed through R2 (0.88), root mean square error (RMSE = 24.08 μg/m3) and mean prediction error (MPE = 16.90 μg/m3). Additionally, SL-XGB also performed the best in the site-based CV with a R2 of 0.86, a RMSE of 26.15 μg/m3 and a MPE of 17.97 μg/m3, which shows its good spatial generalization ability. These results demonstrate that SL-XGB can better simultaneously handle non-linear and spatial heterogeneity issues despite spatially limited data at the urban scale. As far as the PM2.5 concentration distribution was concerned, it presented a gradient increase in PM2.5 concentrations from the northwest to the southeast in Beijing, with abundant spatial details. Overall, the proposed approach for PM2.5 estimation showed outstanding performance and can support preventive pollution control and mitigation at the urban scale. [ABSTRACT FROM AUTHOR]

The authors determined an error in the affiliation section; it was captured as Department of Hematology, Peking Union Hospital, CAMS & PUMC, Beijing 100,730, China. The correct affiliation should be Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing, 100730, China. [ABSTRACT FROM AUTHOR]