Pediatric and adolescent chronic myeloid leukemia: A follow-up study in Western India

Background: Chronic myeloid leukemia (CML) is relatively rare in pediatric and adolescent age groups. The purpose of this study was to evaluate the clinical, hematopathological, and biochemical parameters of CML in pediatric and adolescent age groups, along with an assessment of the treatment response with first-line tyrosine kinase inhibitors (TKI) and its correlation with the prognostic scoring systems of adults. Materials and Methods: A retrospective study of 44 Breakpoint Cluster Region-Abelson leukemia virus (BCR-ABL1)-positive pediatric and adolescent CML cases registered at our hospital was done. The clinical and laboratory parameters were evaluated using hospital software. The treatment response was monitored and scoring was performed using mathematical calculations. Results: The mean age was 11.6 (±4.7) years. The median hemoglobin was 8.4 g/dL and 63.6% of the cases showed white blood cell (WBC) counts >250,000/?L. The average follow-up was 21 months. A total of 97.7 and 78.1% cases achieved complete hematological response (CHR) and molecular response, respectively, during the treatment course. The maximum number of patients had low Sokal and European treatment and Outcomes Study (EUTOS) scores. Seventy-five per cent of the cases achieved CHR at 3 months, while 73.6 and 78.6% CML-Chronic phase (CP) cases with low Sokal and EUTOS scores achieved CHR at 3 months, respectively. Conclusion: This study revealed that the CML cases in pediatric and adolescent age groups are normally present with higher WBC counts at the time of diagnosis. The association of the prognostic scoring system with treatment response was statistically insignificant. However, a larger cohort study is needed to determine the treatment response of TKI in children and adolescent CML and its correlation with the prognostic scoring systems.

Advances in Molecular Targeted Ultrasound Contrast Agents / 中国医学科学院学报

In real-time ultrasound,molecular targeted contrast agent is introduced into the blood circulation through peripheral intravenous injection to enhance the imaging signal of target lesions after binding to the corresponding intravascular receptors,which can realize early diagnosis,staging of diseases,assessment of treatment response,and targeted treatment.In addition,molecular targeted ultrasound contrast agents provide a platform for the delivery of drugs and genes via microbubbles,and nanoscale contrast agents can be infiltrated through vascular endothelium into the interstitial space of the lesion for imaging or treatment.The available studies of molecular targeted ultrasound contrast agents mainly focus on the preclinical trials.Some clinical trials have been conducted in humans and preliminarily confirm the safety and feasibility of targeted ultrasound contrast agents.The molecular targeted ultrasound contrast agents enjoy a broad prospect in clinical application.

Clinical Characteristics and Survival Analysis of Patients with Plasma Cell Leukemia / 中国实验血液学杂志

OBJECTIVE@#To investigate the clinical characteristics, therapeutic response and prognosis of patients with plasma cell leukemia (PCL) and improve the understanding of this disease.@*METHODS@#The clinical manifestations, laboratory tests and treatment response of 27 patients with plasma cell leukemia treated in The Second Hospital of Shanxi Medical University from December 2010 to August 2019 were analyzed retrospectively, and their clinical characteristics were summarized. Kaplan-Meier method was used for survival analysis.@*RESULTS@#There were 18 cases of primary plasma cell leukemia (pPCL) and 9 cases of secondary plasma cell leukemia (sPCL). The male to female ratio was 1.7∶1. The median age was 62 years old. The first manifestations were bone pain, fatigue, fever, splenomegaly and bleeding, and a large number of plasma cell infiltration was observed in the morphological examination of peripheral blood and bone marrow cells. 13 cases were detected by immunotyping and all of them expressed CD38/CD138. 8 cases underwent karyotype analysis, and 3 cases were normal, clonal abnormalities occurred in 5 cases. FISH detection was performed in 12 cases, of which 8 cases were abnormal. In 17 cases of bortezomib based chemotherapy, the ovevall response rate was 52.9%, which was higher than that in the non-bortezomib group, but there was no significant difference between the two groups (P =0.242). The overall median survival time of 27 patients was 6.4 months, the median progression-free survival time was 3.5 months, and the median survival time of patients with pPCL and sPCL was 8.2 months and 2.4 months, respectively, the difference between the two groups was statistically significant (P =0.031).@*CONCLUSION@#PCL is highly invasive and has diverse clinical manifestations, and is not sensitive to traditional chemotherapy. The median survival time of patients with pPCL is relatively longer than that of patients with sPCL. The chemotherapy regimen based on bortezomib improves the treatment effectiveness and prolongs the survival time of PCL patients.

Impact of CSF3R Mutation on Treatment Response and Survival of Patients with Acute Myeloid Leukemia / 中国实验血液学杂志

OBJECTIVE@#To investigate the expression of CSF3R mutation in acute myeloid leukemia (AML) and analyze its clinical characteristics and prognosis.@*METHODS@#A retrospective study was conducted in 212 patients with AML who were newly diagnosed in the Second Hospital of Shanxi Medical University from January 1th 2018 to June 30th 2021, including 22 patients with CSF3R mutations as mutation group and 190 patients with CSF3R wild type ［66 cases of them were screened by propensity score matching (PSM), as control group］. The early efficacy and survival between the two groups were compared.@*RESULTS@#The median age of patients in the mutation group was 50(17-73) years old, and the ratio of male to female was 1.2:1 The main types were AML with maturation (11 cases) and acute myelomonocytic leukemia (9 cases). Prognostic stratification was carried out according to the risk stratification system of the European leukemia network in 2017, with 16 cases (72.73%) in the middle and high-risk group. At the initial diagnosis, the median count of white blood cell (WBC) was 44.75(1.30-368.71)×109/L, among which 15 cases (68.18%) were >10×109/L, and the median count of platelet (PLT) was 24(4-55)×109/L. CSF3R T618I (68.18%) was a common mutation site, which had concomitant gene mutations, in which CEBPA mutation was the most common (10 cases, 45.45%), but only existed in CSF3R T618I mutation. The CR/CRi rate was 68.18% and 71.21% in the mutant group and the control group (P >0.05), the median over all survival time was 15 months and 9 months (P >0.05), and the median disease-free survival time was 8 months and 4 months (P >0.05), respectively.@*CONCLUSION@#Most AML patients with CSF3R mutation are middle-aged patients, the main types are AML with maturation and acute myelomonocytic leukemia, and most of them have middle and high-risk prognosis. CSF3R mutation may not be an independent prognostic marker for newly diagnosed AML patients.

Interferon-related gene array in predicting the efficacy of interferon therapy in chronic hepatitis B / 生物医学工程学杂志

This study aims to clarify host factors of IFN treatment in the treatment of chronic hepatitis B (CHB) patients by screening the differentially expressed genes of IFN pathway CHB patients with different response to interferon (IFN) therapy. Three cases were randomly selected in IFN-responding CHB patients (Rs), non-responding CHB patients (NRs) and healthy participants, respectively. The human type I IFN response RT 2 profiler PCR array was used to detect the expression levels of IFN-related genes in peripheral blood monocytes (PBMCs) from healthy participants and CHB patients before and after Peg-IFN-α 2a treatment. The results showed that more differentially expressed genes appeared in Rs group than NRs group after IFN treatment. Comparing with healthy participants, IFNG, IL7R, IRF1, and IRF8 were downregulated in both Rs and NRs group before IFN treatment; CXCL10, IFIT1, and IFITM1 were upregulated in the Rs; IL13RA1 and IFI35 were upregulated in the NRs, while IFRD2, IL11RA, IL4R, IRF3, IRF4, PYHIN1, and ADAR were downregulated. The expression of IL15, IFI35 and IFI44 was downregulated by 4.09 ( t = 10.58, P < 0.001), 5.59 ( t = 3.37, P = 0.028) and 10.83 ( t = 2.8, P = 0.049) fold in the Rs group compared with the NRs group, respectively. In conclusion, IFN-response-related gene array is able to evaluate IFN treatment response by detecting IFN-related genes levels in PBMC. High expression of CXCL10, IFIT1 and IFITM1 before treatment may suggest satisfied IFN efficacy, while high expression of IL13RA1, IL15, IFI35 and IFI44 molecules and low expression of IFRD2, IL11RA, IL4R, IRF3, IRF4, PYHIN1 and ADAR molecules may be associated with poor IFN efficacy.

Factors predictive of treatment response and survival in Filipino patients with autoimmune hepatitis

Background@#There is a dearth of data on Filipino patients with autoimmune hepatitis (AIH). We aimed to describe the demographic and clinical profiles of patients with AIH and to characterize clinical outcomes and treatment responses.@*Methods@#A retrospective cohort study involving patients from two tertiary centers diagnosed with AIH from January 1, 2007, to December 31, 2019, was included. Disease remission was defined as the normalization of ALT levels, while failure was defined as an increase in ALT levels over baseline or clinical deterioration.@*Results@#A total of 48 patients were identified between 2007 to 2019. The median age at presentation was 51 (27-79 yrs.). Liver cirrhosis was already present in 37.5% (27.1% decompensated) on diagnosis. Aside from a higher histologic activity index in females (p=0.047), there were no gender-specific differences. Disease remission was achieved in 41.9% of patients at 6 months, while only 9.3% failed. At the final disposition, remission rates had slightly increased to 58%, but failure rates had risen to 12%. Treatment responses at both 6 and 12 months and MELD and Child-Pugh class influenced treatment responses at final disposition. Median overall survival was 102 weeks and was influenced by the presence of liver dysfunction and 12 months and final treatment responses.@*Conclusion@#Autoimmune hepatitis remains an important cause of morbidity and mortality. The results of the study highlight the need for immunosuppressive therapy to induce early remission for a higher likelihood of subsequent biochemical remission to reduce the risk of liver-related mortality.

The association between skeletal muscle measures, nutritional risk, cachexia, and chemotherapy-induced toxicity in lung cancer patients / 中华临床营养杂志

Objective:Chemotherapy is the main treatment for lung cancer. However, chemotherapy-related toxicity has a high incidence, which brings adverse experiences to patients and seriously affects treatment plans and quality of life. Low skeletal muscle mass (SMM), malnutrition, and cachexia are related to a higher incidence of chemotherapy toxicity in cancer patients. The main purpose of this study is to evaluate the relationship between baseline body composition, nutritional status, cachexia, and lung cancer chemotherapy toxicity and dose-limiting toxicity (DLT).Methods:This is a single-center prospective study of lung cancer patients receiving chemotherapy for the first time. Body composition was measured using multi-frequency bioelectrical impedance, and the skeletal muscle index (SMI) was calculated. Grip strength and gait speed were used to assess muscle strength and function. The Asian Working Group on Sarcopenia (2019) criteria were used to evaluate the presence of sarcopenia. Cachexia was defined based on muscle mass and weight loss, while nutritional risk and malnutrition were evaluated based on the Nutritional Risk Screening 2002 and Global Leadership Initiative on Malnutrition criteria, respectively. Chemotherapy toxicity and DLT were defined according to the Common Terminology Criteria for Adverse Events and were divided into hematological, non-hematological, and DLT. The association between muscle condition, nutritional status, cachexia, and toxicity was evaluated by univariate and multivariate regression models.Results:The study included and analyzed 126 patients, with a median follow-up of 17.9 months (12-25 months). During chemotherapy, 27.8% of patients experienced grade 3/4 hematological toxicity, 14.8% experienced any ≥2 grade non-hematological toxicity, and 26.1% had any DLT. Old age and multiple comorbidities were risk factors for hematological, non-hematological toxicity, and DLT. Insufficient muscle strength and function could increase the incidence of hematological toxicity [ OR 1.121, 95% CI 1.048~2.865, P=0.031], and high SMI was a protective factor for DLT [ OR 0.638, 95% CI 0.242~0.083, P=0.021]. Conclusions:Lung patients with low SMI, low muscle strength and function have an increased risk of DLT and grade 3/4 hematological toxicity during chemotherapy. At the same time, elderly age and multiple comorbidities are also risk factors for chemotherapy toxicity and DLT. In clinical work, assessments should be conducted to identify high-risk populations for chemotherapy toxicity and preventive measures should be taken. Future research could consider adjusting chemotherapy plans based on different muscle statuses.

Ivermectin treatment response in two rural villages with a high prevalence of onchocerciasis and epilepsy, Mahenge Tanzania

Background: Despite 20 years of ivermectin mass distribution in the Mahenge area, Tanzania, the prevalence of onchocerciasis and epilepsy has remained high in rural villages. Objectives: We investigated the efficacy of ivermectin in reducing Onchocerca volvulus microfilariae and predictors for parasitic load following ivermectin treatment in persons with (PWE) and without epilepsy (PWOE). Methods: Between April and September 2019, 50 PWE and 160 randomly selected PWOE from Msogezi and Mdindo villages participated in a follow-up study. Skin snips were obtained pre (baseline) and three months post-ivermectin treatment. Results: The overall prevalence of O. volvulus positive skin snips at baseline was 49% (103/210), with no significant difference between PWE (58.0%) and PWOE (46.3%); p=0.197. The overall mean micro filarial density was significantly higher at baseline 1.45(95%CI:0.98-2.04)) than three-month post-ivermectin treatment (0.23(95%CI:0.11-0.37), p<0.001. Three months after ivermectin, the micro filarial density had decreased by ≥80% in 54 (81.8%, 95%CI: 72.3-91.4) of the 66 individuals with positive skin snips at baseline. High micro filarial density at baseline was the only significant predictor associated with higher micro filarial density in the post-ivermectin skin snips. Conclusion: Our study reports a decrease in micro filarial density following ivermectin treatment in most individuals. Optimizing ivermectin coverage will address the ongoing onchocerciasis transmission in Mahenge

Advances inbiomarkers for immune thrombocytopenia / 中国输血杂志

Immune thrombocytopenia (ITP) is an autoimmune disease mediated by acquired immunity, with platelets decreased and hemorrhage being the primary clinical manifestations. The responses and platelet count levels after early treatment are important factors affecting long-term prognosis, however, current diagnostic methods and disease evaluation approaches are limited, and lack of specific biomarkers. In recent years, various biomolecules have been proposed as potential biomarkers for ITP. This paper reviews the value and advances in ITP-related biomarkers.

Clinical Role of the Chronic Obstructive Pulmonary Disease Assessment Test in Prediction of the Response to Treatment for Exacerbations

A comparative study on erlotinib & gefitinib therapy in non-small cell lung carcinoma patients

Background & objectives: Tyrosine kinase inhibitors (TKIs) targeting the epidermal growth factor receptor (EGFR) have been evaluated in patients with advanced non-small cell lung cancer (NSCLC). Erlotinib and gefitinib are the first-generation EGFR-TKIs for patients with NSCLC. However, there is a paucity of studies comparing the effectiveness of these two drugs. Hence, this study was aimed to compare the effectiveness and safety of erlotinib and gefitinib in NSCLC patients. Methods: This study included 71 NSCLC patients who received EGFR-TKIs between 2013 and 2016. Adverse drug reaction of both erlotinib (n=37) and gefitinib (n=34) was determined and graded according to Common Terminology Criteria for Adverse Events grading system. Effectiveness was measured using response evaluation criteria in solid tumours and progression-free survival (PFS). Pharmacoeconomic analysis was performed by cost-effective analysis. Results: When comparing safety profile, both the drugs had similar adverse events except for dermal side effects such as acneiform eruption (51.4%), rash (54.05%) and mucositis (59.5%) for erlotinib and 20.6, 26.5 and 29.4 per cent for gefitinib, respectively. The PFS of the two drugs was compared to differentiate the effectiveness of erlotinib and gefitinib. There was no significant difference between the effectiveness of the two drugs. The pharmacoeconomic analysis showed that gefitinib was more cost-effective than erlotinib. Interpretation & conclusions: This study showed that erlotinib and gefitinib had similar effectiveness but gefitinib had a better safety profile compared to erlotinib. Therefore, gefitinib could be considered a better option for NSCLC patients compared to erlotinib. However, further studies need to be done with a large sample to confirm these findings.

Prognostic value of response assessment fluorodeoxyglucose positron emission tomography-computed tomography scan in radically treated squamous cell carcinoma of head and neck: Long-term results of a prospective study

Objective: The objective of this study is to evaluate the diagnostic and prognostic ability of fluorodeoxyglucose positron emission tomography-computed tomography (FDG PET-CT) scan in patients with squamous cell carcinoma of the head and neck treated with chemoradiotherapy or radiotherapy only. Materials and Methods: Fifty-nine patients with HNSCC planned for radical nonsurgical treatment were randomized to receive either three-dimensional conformal radiotherapy or intensity-modulated radiation therapy. In addition to routine clinical examination and staging investigations, patients had a FDG PET-CT scan at baseline and on the first follow-up for response assessment. No evidence of clinicopathological disease for at least 6 months after the completion of treatment was considered confirmation of complete response. The presence or absence of disease during the follow-up period was used to calculate the sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV) of PET-CT for the primary site and node. Results: At a median follow-up of 52.5 months, 55.6% of patients were alive and disease free. Response assessment PET-CT was done at a median of 9 weeks (range: 5–18 weeks). PET-CT assessment of the primary had sensitivity, specificity, PPV, and NPV of 81.8%, 93%, 75%, and 95.2%, respectively; the corresponding figures at the node were 44.4%, 95.6%, 66.7%, and 89.6%. The median baseline maximum standardized uptake value (SUVmax) at primary and node was 14.9 and 8.1, respectively. When PET-CT was done after 10 weeks, no false-positive or false-negative findings were seen. Patients with negative PET at the first follow-up had a significantly better progression-free and overall survival. Conclusions: Disease evaluation using PET-CT has an overall accuracy of 80%. High baseline SUVmax correlates with worse clinical outcomes. Negative PET-CT at the first follow-up is a predictor for survival.

Treating laryngopharyngeal reflux with proton-pump inhibitors - An observational study

Background: Laryngopharyngeal reflux (LPR) refers to the backflow of stomach contents into the throat, that is, into the hypopharynx. LPR is different from classical gastroesophageal reflux disease. Majority of such patients present with globus pharyngeus, cough, foreign body sensation in throat, and hoarseness. Objectives: Conflicting results appear in literature for the role of proton-pump inhibitors (PPIs) in LPR. Our aim in this study is to evaluate the role of PPI based on their effect on reflux finding score (RFS) and reflux symptom index (RSI). Materials and Methods: This prospective observational study was conducted in the Department of ENT and HNS of Government Medical College, Srinagar, for 3 years from 2015 to 2018. The materials for the present study were 70 patients of different age groups attending the ENT Outpatient Department (OPD) having different symptoms of LPR diagnosed on the basis of RFS >7 and RSI >13. Of them, 20 lost to follow up, and finally, 50 patients were followed as per the protocol. Patients were divided into different age groups. Results: A total number of patients included in the study were 50; 35 (70%) cases were females and 15 (30%) were males. A maximum number of patients were in the age group 31–40 years forming about 40% of the study group. Mean RSI of all patients was 24.4 before treatment with PPIs. Significant change in RSI occurred after the first 8 weeks of therapy, and no further significant change occurred in the next 16 weeks. Mean RFS of the patients was 13.2 before treatment with PPIs. There was a slight response after 8 weeks of therapy in physical findings and significant response after 16 weeks of therapy. Conclusion: PPIs are treatment of choice in patients with LPR and treatment should be continued for at least 4 months.

Retrospective analysis of efficacy, safety, and prognostic factors in a cohort of Chinese hepatocellular carcinoma patients treated with drug-eluting bead transarterial chemoembolization

The aim of our study was to assess the efficacy, safety, and prognostic factors of drug-eluting bead transarterial chemoembolization (DEB-TACE) in Chinese hepatocellular carcinoma (HCC) patients. Patients (n=102) diagnosed as primary HCC were consecutively enrolled in this retrospective cohort study. Treatment responses were assessed following the modified Response Evaluation Criteria in Solid Tumors. Progression-free survival (PFS) and overall survival (OS) were evaluated, and adverse events (AEs) as well as liver function-related laboratory indexes of all DEB-TACE records (N=131) were assessed. Complete response (CR) rate, objective response rate, and disease control rate were 51.0, 87.3, and 95.1%, respectively, at 1-3 months post DEB-TACE. The mean PFS and OS were 227 (95%CI: 200-255) days and 343 (95%CI: 309-377) days, respectively. Multivariate logistic regression revealed that portal vein invasion and abnormal total protein (TP) were independent predictive factors for worse CR, and multivariate Cox's regression analysis showed that multifocal disease independently correlated with shorter PFS. Most of the liver function-related laboratory indexes worsened at 1 week but recovered at 1-3 months post-treatment, only the percentage of patients with abnormal ALP increased at 1-3 months. In addition, 112 (85.5%), 84 (64.1%), 53 (40.5%), 40 (30.5%), and 16 (12.2%) patients had pain, fever, nausea, vomiting, and other AEs, respectively. DEB-TACE is efficient and safe in Chinese HCC patients, and portal vein invasion, abnormal TP level as well as multifocal disease could be used as unfavorable prognostic factors to DEB-TACE treatment.

Predictive value of plasma microRNA-126 for treatment response of psoriasis patients to combined treatment with acitretin, methotrexate and ultraviolet phototherapy / 第二军医大学学报

Objective: To investigate the plasma microRNA-126 (miRNA-126) level in psoriasis patients before and after acitretin, methotrexate and ultraviolet phototherapy-combined treatment, and to assess its value in predicting the treatment outcomes. Methods: A total of 196 patients with moderate-to-severe psoriasis were consecutively enrolled and received combined treatment with acitretin, methotrexate and ultraviolet phototherapy, and 200 volunteers were recruited as healthy controls. Plasma samples of psoriasis patients were collected and miRNA-126 level was detected at baseline and after treatment for 1 month, 3 months and 6 months. Psoriasis area and severity index (PASI) were used to assess the disease severity. Treatment response was determined by PASI 50 response (PASI decreased by 50% compared with the baseline) and PASI 75 response (PASI decreased by 75% compared with the baseline). Spearman correlation test was used to analyze the correlation between the expression level of plasma miRNA-126 and PASI. Receiver operating characteristic (ROC) curve was used to evaluate the diagnostic value of relative expression of miRNA-126 in psoriasis patients. Univariate and multivariate logistic regression models were used to analyze the influencing factors of PASI 50 and PASI 75 responses. Results: Baseline miRNA-126 expression was significantly lower in psoriasis patients compared with healthy controls and was negatively correlated with PASI score (r= - 0.222, P=0.002). ROC curve displayed that miRNA-126 had a good diagnostic value for psoriasis (area under curve: 0.700). After treatment for 1 month, 3 months and 6 months, miRNA-126 levels were significantly elevated compared with the baseline (P<0.01). The response rates of PASI 50 were 7.1% (14/196), 37.2% (73/196) and 64.8% (127/196) after treatment for 1 month, 3 months and 6 months, and those of PASI 75 were 1.5% (3/196), 14.3% (28/196) and 35.7% (70/196), respectively. PASI 50 and PASI 75 responders presented lower baseline miRNA-126 level (P<0.05). Logistic regression analysis revealed that baseline miRNA-126 level was negatively associated with PASI 50 and PASI 75 responses. Conclusion: Plasma miRNA-126 level gradually increases in psoriasis patients after acitretin, methotrexate and ultraviolet phototherapy-combined treatment, and the baseline miRNA-126 level is negatively correlated with treatment response.

Impact of solid tumor history on treatment response and survival of patients with diffuse large B-cell lymphoma / 中华血液学杂志

Objective@#To observe whether the history of solid tumors affects the treatment response and survival situation of patients with diffuse large B-cell lymphoma (DLBCL) .@*Methods@#A retrospective study was conducted in 836 patients with DLBCL who were treated in the Department of Hematology at Ruijin Hospital from 2013 to 2018. Among them, 34 DLBCL patients who had the history of solid tumors were classified into double cancer group. From 802 patients without history of solid tumors, 68 DLBCL patients were selected as control group, using 1∶2 matching on propensity scores for age, gender, IPI score and etc. All patients included in the study had follow-up interviews through medical record and telephone for mortality from any cause. Treatment response and 3-year overall survival (OS) and progression-free survival (PFS) of two groups were analyzed.@*Results@#The complete remission rates after RCHOP (Rituximab+Cyclophosphamide+Vincristine+Adriamycin 50 mg/m2 or Epirubicin or Liposome Adriamycin+Prednisone) regimen were 79.4% and 67.6% in the double cancer group and the control group, respectively (P=0.210) . Among the 102 patients, 6 patients died in the double cancer group while 24 patients died in the control group and the median survival time of both two groups were not reached. The 3-year OS were (74.7±9.5) % and (63.5±6.1) % (χ2=2.791, P=0.095) , while 3-year PFS were (72.1±8.8) % and (54.3±6.4) % (χ2=1.400, P=0.237) in the double cancer group and the control group, respectively.@*Conclusion@#The history of solid tumors didn’t affect DLBCL patients’ treatment response and short-term survival.

Analysis of clinical characteristics, treatment response rate and survival of 77 myelodysplastic syndrome patients with del (5q) syndrome / 中华血液学杂志

Objective@#To observe the clinical characteristics, treatment responses and prognosis of patients with myelodysplastic syndrome (MDS) -del (5q) syndrome who met WHO (2016) diagnostic typing criteria.@*Methods@#A total of 77 patients with del (5q) syndrome, according to WHO (2016) classification, were retrospectively analyzed between January 2008 and April 2018 in the Blood Diseases Hospital, Chinese Academy of Medical Sciences. Clinical characteristics, lenalidomide (LEN) efficacy and survivals were compared between the patients with del (5q) alone and those with one additional cytogenetic abnormality (ACA) with the exception of monosomy 7 or del (7q) . Treatment response and overall survival (OS) were compared between patients who were treated with LEN and traditional non-LEN drugs.@*Results@#Of 77 patients, 64 were isolated del (5q) and 13 were del (5q) with ACA. There were significant differences of the median age and percentage of patients who had small megakaryocytes in bone marrow smear by immunohistochemistry (CD41) between the patients with isolated del (5q) and the patients with del (5q) + ACA[58 (29-64) years old vs 63 (31-82) years old, z=2.164, P=0.030; and 91.7%vs 60.0%, P=0.046, respectively]. The overall hematological response rate (78.9%vs 80.0%) , complete hematological remission (CR) rate (57.9% vs 60.0%) , cytogenetic response (CyR) rate[69.2% (9/13) vs 66.7% (4/6) ] and complete cytogenetic response (CCyR) rate [61.5% (8/13) vs 33.3% (2/6) ] of LEN were similar between the patients with isolated del (5q) (n=19) and with del (5q) + ACA (n=10) , as well as the median Overall survival (OS) between these two groups of patients (62 months vs 78 months, P=0.388) . The hematological response rate (79.3% vs 36.0%) , CR rate (58.6% vs 8.0%) , CyR rate [68.4% (13/19) vs 11.1% (1/9) ] and CCyR rate [52.6% (10/19) vs 0 (0/9) ] were higher among patients treated with LEN (n=29) than those treated with non-LEN therapy (n=25) . There was no statistically significant difference in OS between the patients with LEN or non-LEN therapy (78 months vs 62 months, P=0.297) .@*Conclusion@#Comparing del (5q) syndrome patients with isolated del (5q) or with del (5q) + ACA, two groups of patients had similar clinical characteristics, median OS and LEN efficacy. LEN showed better treatment response than traditional drugs in patients with del (5q) syndrome.

Influence of Rome Ⅳ diagnostic criteria on patients with irritable bowel syndrome / 中华消化杂志

Objective To investigate the improvement of symptoms of the patients after treatment in patients with Rome Ⅳ or non-Rome Ⅳ irritable bowel syndrome (IBS),and to explore the influence of IBS diagnosed by different criteria on the patients.Methods From June 2nd to 8th in 2016,at Outpatients Department of Gastroenterology,Union Hospital Affiliated to Tongji Medical College,Huazhong Uiversity of Science and Technology in Wuhan,1 500 outpatients aged over 18 years old and with intestinal symptom were selected for questionnaire.After treatment for six months,IBS patients,non-IBS patients,patients with Rome Ⅳ IBS and patients with non-Rome Ⅳ IBS were followed up by phone calls.After treatment,the improvement of symptoms of the patients was evaluated by irritable bowel syndrome symptom severity scale (IBS-SSS).The degree of influence of IBS diagnosed with different criteria on patients was evaluated by the patient's daily work whether to choose colonoscopy examination,whether to choose medication,and the efficacy of medicine.Student's t test,Mann-Whitney U test and chi-square test were performed for statistical analysis.Results A total of 352 patients with intestinal symptoms were followed-up,including 175 patients with IBS (84 patients with Rome Ⅳ IBS and 91 patients with non-Rome Ⅳ IBS) and 177 non-IBS patients,and 142 patients responded.There were no statistically significant differences in response rate between non-IBS patients and IBS patients (37.3％,66/177 vs.43.4％,76/175),and between patients with Rome Ⅳ IBS and patients with non-Rome Ⅳ IBS (40.5％,34/84 vs.46.2％,42/91) (x2 =1.379 and 0.573,P =0.240 and 0.449).Compared with the non-IBS patients,the degree of satisfaction of medicine was lower in IBS patients (71.4％,30/42 vs.47.5％,19/40).Compared with non-Rome Ⅳ IBS patients,Rome type Ⅳ IBS patients were more likely to receive colonoscopy (35.7％,15/42 vs.58.8％,20/34),and the differences were statistically significant (x2 =4.878 and 4.039,P =0.027 and 0.044).After six months of treatment,symptoms improved in both Rome Ⅳ IBS patients and non-Rome Ⅳ IBS patients (both P ＜ 0.05),however,the symptoms improved more significantly in Rome Ⅳ IBS patients and the total score of IBS-SSS was lower than that of non-Rome Ⅳ IBS patients (-130,-185 to 60 vs.-70,-100 to 28),and the difference was statistically significant (Z =-3.065,P =0.002).The difference was mainly showed the symptom of abdominal pain,and the IBS-SSS abdominal pain score of Rome Ⅳ IBS patients was lower than that of non-Rome Ⅳ IBS patients (-80,-100 to-40 vs.0,-40 to 0),and the difference was statistically significant (Z =-4.631,P ＜ 0.01).Conclusions IBS symptoms influence a lot on the satisfaction degree of treatment in outpatients.Even with similar good therapeutic effects,the Rome Ⅳ IBS symptoms have a more severe impact on patients than non-Rome Ⅳ IBS symptoms.

Clinical and Neuropsychological Factors Associated with Treatment Response and Adverse Events of Atomoxetine in Children with Attention-Deficit/Hyperactivity Disorder

OBJECTIVES: The objective of this study was to investigate clinical and neuropsychological factors associated with treatment response and adverse events of atomoxetine in children with attention-deficit/hyperactivity disorder (ADHD) in Korea. METHODS: Children with ADHD were recruited at the Department of Psychiatry of Asan Medical Center from April 2015 to April 2018. Diagnoses of ADHD and comorbid psychiatric disorders were confirmed with the Kiddie-Schedule for Affective Disorders and Schizophrenia-Present and Lifetime Version. The subjects were subsequently treated with atomoxetine for 12 weeks and illness severity was scored using the ADHD Rating Scale, Clinical Global Impression-Severity scale (CGI-S) and/or Improvement scale (CGI-I), at pre- and post-treatment. They also completed the Advanced Test of Attention (ATA), while their caregivers completed the Korean Personality Rating Scale for Children (KPRC) at pre- and post-treatment. Independent t-test, Fisher's exact test, χ2 test, mixed between-within analysis of variance and correlation analysis were used for statistical analysis. RESULTS: Sixty-five children with ADHD (mean age: 7.9±1.4 years, 57 boys) were enrolled, of which, 33 (50.8%) were treatment responders. Scores on the social dysfunction subscale of the KPRC (p=0.021) and commission errors on the visual ATA (p=0.036) at baseline were higher in treatment non-responders than in responders; however, the statistical significances disappeared after adjusting for multiple comparisons. Mood changes were also observed in 13 subjects (20.0%), and three of them discontinued atomoxetine due to this. Additionally, atomoxetine-emergent mood change was observed more frequently in girls (p=0.006), while the intelligence quotient (p=0.040) was higher in those subjects with mood changes than in those without. CONCLUSION: The results of our study suggest that clinical and neuropsychological factors could be associated with treatment response or adverse events of atomoxetine in children with ADHD. Further long-term studies with larger samples are needed.

Development of Web-Based Nomograms to Predict Treatment Response and Prognosis of Epithelial Ovarian Cancer / Journal of the Korean Cancer Association, 대한암학회지

PURPOSE: Discovery of models predicting the exact prognosis of epithelial ovarian cancer (EOC) is necessary as the first step of implementation of individualized treatment. This study aimed to develop nomograms predicting treatment response and prognosis in EOC. MATERIALS AND METHODS: We comprehensively reviewed medical records of 866 patients diagnosed with and treated for EOC at two tertiary institutional hospitals between 2007 and 2016. Patients’ clinico-pathologic characteristics, details of primary treatment, intra-operative surgical findings, and survival outcomes were collected. To construct predictive nomograms for platinum sensitivity, 3-year progression-free survival (PFS), and 5-year overall survival (OS), we performed stepwise variable selection by measuring the area under the receiver operating characteristic curve (AUC) with leave-one-out cross-validation. For model validation, 10-fold cross-validation was applied. RESULTS: The median length of observation was 42.4 months (interquartile range, 25.7 to 69.9 months), during which 441 patients (50.9%) experienced disease recurrence. The median value of PFS was 32.6 months and 3-year PFS rate was 47.8% while 5-year OS rate was 68.4%. The AUCs of the newly developed nomograms predicting platinum sensitivity, 3-year PFS, and 5-year OS were 0.758, 0.841, and 0.805, respectively. We also developed predictive nomograms confined to the patients who underwent primary debulking surgery. The AUCs for platinum sensitivity, 3-year PFS, and 5-year OS were 0.713, 0.839, and 0.803, respectively. CONCLUSION: We successfully developed nomograms predicting treatment response and prognosis of patients with EOC. These nomograms are expected to be useful in clinical practice and designing clinical trials.