Simultaneous quantification of six proteins related to liver injury using nano liquid chromatography-tandem mass spectrometry.

RATIONALE: In clinical diagnosis of liver injury, which is an important health concern, serum aminotransferase assays have been the go-to method used worldwide. However, the measurement of serum enzyme activity has limitations, including inadequate disease specificity and enzyme specificity. METHODS: With the high selectivity and specificity provided by nano liquid chromatography-tandem mass spectrometry (LC/MS/MS), this work describes a method for the simultaneous determination of six proteins in liver that can be potentially used as biomarkers for liver injury: glutamic-pyruvic transaminase 1 (GPT1), glutamic oxaloacetic transaminase 1 (GOT1), methionine adenosyl transferase 1A (MAT1A), glutathione peroxidase 1 (GPX1), cytokeratin 18 (KRT18) and apolipoprotein E (APOE). RESULTS: In validation, the method was shown to have good selectivity and sensitivity (limits of detection at pg/mL level). The analytical method revealed that, compared with normal mice, in carbon tetrachloride-induced acute liver injury mice, liver MAT1A and GPX1 were significantly lower (p < 0.01 and p < 0.05, respectively), KRT18 was significantly higher (p < 0.05) and APOE and GPT1 were marginally significantly lower (p between 0.05 and 0.1). This is the first work reporting the absolute contents of GPT1, GOT1, MAT1A, GPX1 and KRT18 proteins based on LC/MS. CONCLUSIONS: The proposed method provides a basis for establishing more specific diagnostic indicators of liver injury.

Develop prediction model to help forecast advanced prostate cancer patients' prognosis after surgery using neural network.

Background: The effect of surgery on advanced prostate cancer (PC) is unclear and predictive model for postoperative survival is lacking yet. Methods: We investigate the National Cancer Institute's Surveillance, Epidemiology, and End Results (SEER) database, to collect clinical features of advanced PC patients. According to clinical experience, age, race, grade, pathology, T, N, M, stage, size, regional nodes positive, regional nodes examined, surgery, radiotherapy, chemotherapy, history of malignancy, clinical Gleason score (composed of needle core biopsy or transurethral resection of the prostate specimens), pathological Gleason score (composed of prostatectomy specimens) and prostate-specific antigen (PSA) are the potential predictive variables. All samples are divided into train cohort (70% of total, for model training) and test cohort (30% of total, for model validation) by random sampling. We then develop neural network to predict advanced PC patients' overall. Area under receiver operating characteristic curve (AUC) is used to evaluate model's performance. Results: 6380 patients, diagnosed with advanced (stage III-IV) prostate cancer and receiving surgery, have been included. The model using all collected clinical features as predictors and based on neural network algorithm performs best, which scores 0.7058 AUC (95% CIs, 0.7021-0.7068) in train cohort and 0.6925 AUC (95% CIs, 0.6906-0.6956) in test cohort. We then package it into a Windows 64-bit software. Conclusion: Patients with advanced prostate cancer may benefit from surgery. In order to forecast their overall survival, we first build a clinical features-based prognostic model. This model is accuracy and may offer some reference on clinical decision making.

A Primary Report 166 Cases of Abdominal or Pelvic Neuroblastoma Surgery Utilizing the International Neuroblastoma Surgical Report Form (INSRF).

BACKGROUND AND AIMS: Surgery is pivotal in the management of neuroblastoma (NB), particularly in patients with Image-Defined Risk Factors (IDRFs). The International Neuroblastoma Surgical Report Form (INSRF) was introduced to enhance surgical reporting quality and analyze the defining role of extensive surgery in NB. This study reports our experience with INSRF and explores new criteria for evaluating the extent of surgical resection. METHODS: INSRF was deployed to critically analyze 166 patients with abdominal or pelvic NB who underwent surgery at our department between October 2021 and June 2023. Patient demographics, clinical characteristics, surgical datasets, and postoperative complications were described in detail. Receiver operating characteristic (ROC) curves were used to explore a new method to evaluate the extent of resection. A questionnaire was formulated to obtain attitudes/feedback and commentary from surgical oncologists with INSRF. RESULTS: 166 neuroblastoma patients with a median disease age 36.50 months. This study collated 320 INSRF reports. Among the 166 index cases, 137 were documented by two surgeons, with a concordance rate of 16.78%. Items with high inconsistency were (i) the extent of tumor resection (29.20%), (ii) renal vein involvement (25.55%), (iii) abdominal aorta encasement (16.79%), and (iv) mesenteric infiltration (17.52%). According to INSRF, the extent of resection was complete excision in 86 (51.81%) patients, minimal residual tumor < 5 cm3 in 67 (40.36%) patients, and incomplete excision > 5 cm3 in 13 (7.83%) patients. In ROC curve analysis, the number of vessels encased by tumors > 3 had a high predictive value in determining that a tumor could not be completely resected (AUC 0.916, sensitivity 0.838, specificity 0.826) using INSRF as the gold standard reference. The questionnaires showed that surgeons agreed that the extent of resection and tumor involvement of organ/vascular structures were important, while the definition and intervention(s) of intraoperative complications were less operational and understandable. CONCLUSIONS: INSRF has significant clinical application in neuroblastoma surgery. The extent of resection can be predicted based on the number of tumor-encased blood vessels. Supplementary information should be considered with the INSRF to aid practitioner reporting. Multicenter studies are needed to explore the defining role of INSRF in NB surgical management.

Development of the pediatric family-based dignity therapy protocol for terminally ill children (ages 7-18) and their families: A mixed-methods study.

OBJECTIVES: Dignity therapy (DT) is well-established in adults, and it might potentially benefit the younger population. This study aims to develop a pediatric family-based dignity therapy (P-FBDT) protocol for terminally ill children and their families. METHODS: A parallel mixed-methods design was used. The P-FBDT protocol was developed based on the adult DT, and meanwhile by taking children-specific dignity characteristics and Chinese family-oriented culture into consideration. The protocol was then evaluated and modified based on the quantitative and qualitative feedback from 2-round surveys of 14 pediatric oncology or pediatric palliative care experts. RESULTS: The P-FBDT involves terminally ill children and their families in meaningful interactions including a series of conversations and creative activities, which will be recorded and then edited into a document-based generativity entity. The P-FBDT protocol was recognized as highly reasonable and the P-FBDT interview guide was endorsed as important, acceptable, clear, comprehensive, and suitable to be used in pediatric palliative care practice in Chinese culture (>90%). Potential benefits, possible challenges, and practical considerations of the P-FBDT were also proposed. SIGNIFICANCE OF RESULTS: The P-FBDT was perceived to be potentially beneficial to terminally ill children and their families by engaging in a series of meaningful family interactions and creating a lasting memento to be preserved. The protocol needs to be pilot tested among terminally ill children and families for feasibility and potential efficacy in practice.

In Every Detail: Spiritual Care in Pediatric Palliative Care Perceived by Healthcare Providers.

CONTEXT: Spiritual care is an essential domain of pediatric palliative care. The current mainland China faces a lack of national guidance and a shortage of specialized personnel to provide spiritual care in a traditional developing country. OBJECTIVES: To identify spiritual care in pediatric palliative care services in mainland China from the perspective of healthcare professionals. METHODS: A qualitative descriptive interview study was conducted individually with 27 participants: 14 physicians, seven nurses, and six social workers. The data were analyzed using thematic analysis. RESULTS: Participants described that the essence of spiritual support was provided "in every detail" throughout pediatric palliative care. Four major themes and eleven subthemes were identified. 1) Assessing spiritual needs: paying attention to different perspectives; considering religion, tradition, and culture; discovering spiritual needs behind other needs. 2) Facilitating spiritual exploration: being with the family; providing resources; guiding by providers' own faith; 3) Supporting connections: encouraging the building of personal bonds; facilitating the establishment of spiritual connections. 4) Relieving spiritual suffering: facilitating a family review of child's life; supporting building meaning in daily life; assisting in leaving a legacy for the child. CONCLUSION: This study illustrated that current spiritual support, though not formally organized, is provided individually in pediatric palliative care services in mainland China. Strategies for a practice guide, education and training for professionals, and cultural building need to be rationally developed to strengthen and structure spiritual support integrated into pediatric palliative care.

A shared decision-making model in pediatric palliative care: a qualitative study of healthcare providers.

BACKGROUND: Pediatric shared decision-making (SDM) is a fundamental part of family-centered care. Pediatric palliative care (PPC) is one of the more difficult fields for healthcare providers when choosing to utilize SDM. However, to our knowledge, there are still few structured approaches of SDM in PPC. We aimed to build a model of SDM in PPC that achieves better care and outcomes for children and their family members. METHODS: This study is a descriptive phenomenology study. Participants included physicians, nurses, and social workers in the PPC team. Participants were individually interviewed face-to-face or via an online meeting software. Data were collected in semi-structured interviews and analyzed using a thematic framework analysis. RESULTS: In total, 27 healthcare providers were interviewed. The model of SDM in PPC identified three themes, including the participants, the principle and the process of SDM. Decision participants involved the children, parents, the PPC team and others. The decision principle had three sub-themes including type, standard and precondition. The decision process describes the fundamental process of SDM and provides suggestions for mobilizing patients and parents to engage in decision-making and seeking conflict resolution. CONCLUSIONS: This is the first study to develop a SDM model in PPC. This model can provide guidance to PPC teams on SDM practices. In addition, the model contributes to the existing body of knowledge by providing a conceptual model for SDM in the context of PPC.

A deep learning-based model (DeepMPM) to help predict survival in patients with malignant pleural mesothelioma.

Background: Malignant pleural mesothelioma (MPM) is a rare disease with limited treatment and poor prognosis, and a precise and reliable means to predicting MPM remains lacking for clinical use. Methods: In the population-based cohort study, we collected clinical characteristics from the Surveillance, Epidemiology, and End Results (SEER) database. According to the time of diagnosis, the SEER data were divided into 2 cohorts: the training cohort (from 2010 to 2016) and the test cohort (from 2017 to 2019). The training cohort was used to train a deep learning-based predictive model derived from DeepSurv theory, which was validated by both the training and the test cohorts. All clinical characteristics were included and analyzed using Cox proportional risk regression or Kaplan-Meier curve to determine the risk factors and protective factors of MPM. Results: The survival model included 3,130 cases (2,208 in the training cohort and 922 in the test cohort). As for model's performance, the area under the receiver operating characteristics curve (AUC) was 0.7037 [95% confidence interval (CI): 0.7030-0.7045] in the training cohort and 0.7076 (95% CI: 0.7067-0.7086) in the test cohort. Older age; male sex, sarcomatoid mesothelioma; and T4, N2, and M1 stage tended to be the risk factors for survival. Meanwhile, epithelioid mesothelioma, surgery, radiotherapy, and chemotherapy tended to be the protective factors. The median overall survival (OS) of patients who underwent surgery combined with radiotherapy was the longest, followed by those who underwent a combination of surgery, radiotherapy, and chemotherapy. Conclusions: Our deep learning-based model precisely could predict the survival of patients with MPM; moreover, multimode combination therapy might provide more meaningful survival benefits.

Data-Driven Tool for Cross-Run Ion Selection and Peak-Picking in Quantitative Proteomics with Data-Independent Acquisition LC-MS/MS.

Proteomics provides molecular bases of biology and disease, and liquid chromatography-tandem mass spectrometry (LC-MS/MS) is a platform widely used for bottom-up proteomics. Data-independent acquisition (DIA) improves the run-to-run reproducibility of LC-MS/MS in proteomics research. However, the existing DIA data processing tools sometimes produce large deviations from true values for the peptides and proteins in quantification. Peak-picking error and incorrect ion selection are the two main causes of the deviations. We present a cross-run ion selection and peak-picking (CRISP) tool that utilizes the important advantage of run-to-run consistency of DIA and simultaneously examines the DIA data from the whole set of runs to filter out the interfering signals, instead of only looking at a single run at a time. Eight datasets acquired by mass spectrometers from different vendors with different types of mass analyzers were used to benchmark our CRISP-DIA against other currently available DIA tools. In the benchmark datasets, for analytes with large content variation among samples, CRISP-DIA generally resulted in 20 to 50% relative decrease in error rates compared to other DIA tools, at both the peptide precursor level and the protein level. CRISP-DIA detected differentially expressed proteins more efficiently, with 3.3 to 90.3% increases in the numbers of true positives and 12.3 to 35.3% decreases in the false positive rates, in some cases. In the real biological datasets, CRISP-DIA showed better consistencies of the quantification results. The advantages of assimilating DIA data in multiple runs for quantitative proteomics were demonstrated, which can significantly improve the quantification accuracy.

Predicting cutaneous malignant melanoma patients' survival using deep learning: a retrospective cohort study.

BACKGROUND: Cutaneous malignant melanoma (CMM) has the worst prognosis among skin cancers, especially metastatic CMM. Predicting its prognosis accurately could direct clinical decisions. METHODS: The Surveillance, Epidemiology, and End Results database was screened to collect CMM patients' data. According to diagnosed time, patients were subdivided into three cohorts, train cohort (diagnosed between 2010 and 2013), validation cohort (diagnosed in 2014), and test cohort (diagnosed in 2015). Train cohort was used to train deep learning survival model for cutaneous malignant melanoma (DeepCMM). DeepCMM was then evaluated in train cohort and validation cohort internally, and validated in test cohort externally. RESULTS: DeepCMM showed 0.8270 (95% CI, confidence interval, CI 0.8260-0.8280) as area under the receiver operating characteristic curve (AUC) in train cohort, 0.8274 (95% CI 0.8286-0.8298) AUC in validation cohort, and 0.8303 (95% CI 0.8289-0.8316) AUC in test cohort. Then DeepCMM was packaged into a Windows 64-bit software for doctors to use. CONCLUSION: Deep learning survival model for cutaneous malignant melanoma (DeepCMM) can offer a reliable prediction on cutaneous malignant melanoma patients' overall survival.

Neural network-based prognostic predictive tool for gastric cardiac cancer: the worldwide retrospective study.

BACKGROUNDS: The incidence of gastric cardiac cancer (GCC) has obviously increased recently with poor prognosis. It's necessary to compare GCC prognosis with other gastric sites carcinoma and set up an effective prognostic model based on a neural network to predict the survival of GCC patients. METHODS: In the population-based cohort study, we first enrolled the clinical features from the Surveillance, Epidemiology and End Results (SEER) data (n = 31,397) as well as the public Chinese data from different hospitals (n = 1049). Then according to the diagnostic time, the SEER data were then divided into two cohorts, the train cohort (patients were diagnosed as GCC in 2010-2014, n = 4414) and the test cohort (diagnosed in 2015, n = 957). Age, sex, pathology, tumor, node, and metastasis (TNM) stage, tumor size, surgery or not, radiotherapy or not, chemotherapy or not and history of malignancy were chosen as the predictive clinical features. The train cohort was utilized to conduct the neural network-based prognostic predictive model which validated by itself and the test cohort. Area under the receiver operating characteristics curve (AUC) was used to evaluate model performance. RESULTS: The prognosis of GCC patients in SEER database was worse than that of non GCC (NGCC) patients, while it was not worse in the Chinese data. The total of 5371 patients were used to conduct the model, following inclusion and exclusion criteria. Neural network-based prognostic predictive model had a satisfactory performance for GCC overall survival (OS) prediction, which owned 0.7431 AUC in the train cohort (95% confidence intervals, CI, 0.7423-0.7439) and 0.7419 in the test cohort (95% CI, 0.7411-0.7428). CONCLUSIONS: GCC patients indeed have different survival time compared with non GCC patients. And the neural network-based prognostic predictive tool developed in this study is a novel and promising software for the clinical outcome analysis of GCC patients.

The dignity of terminally ill children in pediatric palliative care: perspectives of parents and healthcare providers.

BACKGROUND: The Chochinov Dignity Model was developed based on a cohort of adult patients with advanced cancer, but its role among dying children is not clear. This study aims to develop a model of dignity for children receiving pediatric palliative care based on the Chochinov Dignity Model. METHODS: This is a descriptive qualitative study. Participants included a total of 11 parents and 14 healthcare providers who were recruited from a tertiary children's hospital in Beijing and the Pediatric Palliative Care Subspecialty Group of the Pediatrics Society of the Chinese Medical Association using purposive sampling. Thematic framework analysis was used to analyze data. RESULTS: The themes of the empirical Dignity Model were broadly supported in this study, but some themes were interpreted differently in the child population. Compared with the original model, some child-specific themes were identified including acknowledging regret, a sense of security, the company of important loved ones, realizing unfinished wishes, decent and dignified death, resolving family disputes, and fairness. CONCLUSIONS: This is the first study on Dignity Model for terminal children. Knowledge of children's dignity can promote reflection of healthcare providers and caregivers regarding the values underlying their performance in pediatric palliative care, and develop certain practical interventions to strengthen children and their families' sense of dignity at end of life.

Acquired CFTR dysfunction and dense distribution of ionocytes in nasal mucosa of children with CRS.

BACKGROUND: Ionocytes are rare cells in airway epithelium characterized by a high expression of CFTR. OBJECTIVES: To investigate the morphology and distribution of ionocytes and the function of CFTR in the nasal mucosal epithelium of children. METHODS: The exfoliated cells of nasal mucosa from 101 children were detected using flow cytometry to analyze the number of ionocytes and CFTR and the difference of CFTR function. Nasal mucosa and polyps were collected from 10 children with CRSwNP. The RNAscope of FOXI1 and CFTR was detected in pathological paraffin sections. The expression and distribution of ionocytes and CFTR in nasal mucosa and polyp epithelium were observed. RESULTS: In CRS patients, the number of ionocytes in the nasal epithelium was lower and the number of ionocytes that did not express CFTR was higher, and the function of CFTR was also decreased. The expression of CFTR in the nasal mucosa of CRS showed the characteristics of local dense distribution and increased as the inflammation expanded. The ionocytes were "tadpole-shaped" in the epithelium and gathered in the area of high CFTR expression, the intracellular CFTR was expanded in clusters. Ionocytes that did not express CFTR was more common in the nasal polyps. CONCLUSIONS: The number of ionocytes and the function of CFTR in nasal mucosa of CRS patients decreased. With the expansion of inflammation, CFTR and ionocytes showed more obvious dense distribution. Some ionocytes lost the expression of CFTR and did not show the "tadpole" shape, which may be related to the occurrence of polyps.

Pain management of newly diagnosed sarcoma patients at a single center.

BACKGROUND: Systematic pain management of children is insufficient in China, and there is no literature on pain in children with sarcoma. METHODS: Clinical data of 188 patients with newly diagnosed sarcoma admitted to the Medical Oncology Department of Beijing Children's Hospital was collected from October 2018 to December 2020. Children experiencing pain received analgesic treatment and regular assessment. RESULTS: Thirty-seven patients (19.7%) suffered from pain. Six cases (16.2%) had mild pain, 17 (46.0%) moderate, and 14 (37.8%) severe. Daily lives of 31 patients were affected by pain. Twenty-six cases had bone invasion. The analgesic rate was 54.1% before admission and 89.2% after admission. Nine cases were treated with oral morphine regularly, and their pain was relieved before chemotherapy; the dose of morphine was 0.14 ±â€…0.034 mg/kgQ4H when the target was reached. No serious adverse reactions were observed. The period of morphine application after chemotherapy was 5 to 9 days, and there was no withdrawal reaction. CONCLUSION: Pain in children with newly diagnosed sarcoma was mainly moderate to severe, and the incidence of pain in sarcoma with bone invasion was higher, with greater intensity. Patients who received standardized pain assessment and regular analgesics reached pain relief quickly, and no serious adverse reactions were observed within the recommended dosage.

Development levels of pediatric palliative care teams and the extent of palliative care understanding and implementation among pediatric oncologists in China.

IMPORTANCE: Pediatric palliative care (PPC) is an interdisciplinary collaboration that focuses on the prevention and relief of patient suffering. PPC has emerged as a critical field of medical expertise and practice. However, no information is available regarding the progress of PPC in the Chinese mainland. OBJECTIVE: This study investigated the geographic distribution, team structure, and services of PPC teams in the Chinese mainland. It also investigated the level of understanding and implementation among pediatric oncologists regarding PPC. METHODS: The PPC subspecialty group of the Pediatrics Society of the Chinese Medical Association included 45 PPC teams. The team structure and services were investigated using questionnaires mailed to the team leader of each PPC team. In addition, we sent questionnaires regarding the level of PPC understanding and implementation of PPC practices to 170 pediatric oncologists in 11 hospitals. RESULTS: The geographical distribution of PPC teams is uneven in China. Most PPC teams are concentrated in the eastern provincial capital of China. Most PPC teams had limited staff and services. The level of PPC understanding was considerably limited across all demographics; most pediatric oncologists reported "some understanding" (n = 71, 41.8%) or "poor understanding" (n = 50, 29.4%). Only 62.9% of pediatric oncologists had experience providing advice to family members regarding PPC matters. INTERPRETATION: China is currently experiencing a critical shortage of PPC resources. Most pediatric oncologists had a limited understanding of PPC and reported limited practical implementation of PPC, which leads to underutilization of PPC resources.

The development and initial evaluation of referral flowchart for suspected neuroblastoma for pediatricians in nononcology clinics in China.

BACKGROUND: The delayed diagnosis of neuroblastoma (NB) is common in China, which results in the prognosis of NB in China lagging behind that in developed countries. METHODS: A referral flowchart for suspected NB was implemented in nononcology clinics at Beijing Children's Hospital (BCH). Patients with symptoms of suspected NB were referred from nononcology clinics in BCH to oncology clinics and confirmed NB cases were regarded as referral group. The control group comprised patients initially diagnosed with NB who came directly to oncology clinics in BCH from other regions nationwide. The age at NB diagnosis was compared as primary outcome, and the 5-year overall survival (OS) and event-free survival (EFS) were compared via the Kaplan-Meier method and log-rank tests. RESULTS: In total, 3337 children with suspected NB were screened consecutively from 687 070 pediatric patients. Through examination of urine vanillylmandelic acid and homovanillic acid, or B-ultrasound, 102 of 3337 patients were referred to oncologists for comprehensive evaluations. Eventually, 29 referred patients were diagnosed as NB and the hospital-based diagnosis rate of NB was 4.2 per 100 000 visits. The median age at diagnosis in the referral group was 21.0 months, which was 9 months earlier than that of the control group (30.0 months, P = .026). The 5-year OS rate was 72.4% in the referral group, which was higher than that of the control group (66.7%) but without statistical significance (P = .664). CONCLUSION: Delayed NB detection could be avoided by training pediatricians in nononcology clinics to detect suspected NB and refer these patients to oncologists.

Risk Factors and Reasons for Discharge Against Medical Advice for Newborns With Neonatal Surgical Diseases: A Preliminary Study From a Tertiary Care Hospital in Beijing, China.

Background: To identify the risk factors and reasons for discharge against medical advice (DAMA) for newborns with neonatal surgical diseases in a tertiary care hospital in China. Methods: A retrospective study was conducted on all newborn patients admitted to the neonatal surgery department of Beijing Children's Hospital between January 1, 2016 and January 1, 2020. Medical records were compared between DAMA and non-DAMA patients. Univariate and multivariate logistic regression analyses were conducted to identify potentially useful characteristics for predicting DAMA. Results: During the study period, 854 newborns were admitted to the neonatal surgery department. A total of 68 DAMA patients (68/854, 7.96%, 47 boys), with a median age at diagnosis of 1 day (range, from birth to 21 days), were included in this study. After multivariate analysis, we found that emergency admission, age at admission ≤5 days, rejection for surgery, and admission to the neonatal intensive care unit were significant independent risk factors for DAMA. According to the electronic medical records, the reasons for DAMA included belief in incurability and concerns about the prognosis of the disease (n = 31), multiple malformations with poor prognosis (n = 8), severe postoperative complications (n = 5), financial difficulties (n = 3), refusal of further examinations (n = 2), assumption of clinical improvement (n = 1), and unknown (n = 18). Conclusions: This preliminary study showed that neonatal surgical patients in critical conditions were high-risk groups for DAMA, and the main possible reasons for DAMA were the parents' belief in incurability and concerns about the prognosis of the disease.

Outcome of two pairs of monozygotic twins with pleuropulmonary blastoma: case report.

BACKGROUND: Pleuropulmonary blastomas (PPB) are rare aggressive paediatric lung malignancies and are among the most common DICER1-related disorders: it is estimated that 75-80% of children with a PPB have the DICER1 mutation. DICER1 mutations are responsible for familial tumour susceptibility syndrome with an increased risk of tumours. In approximately 35% of families with children manifesting PPB, further malignancies may be observed. Symptoms of DICER1 syndrome may vary, even within monozygotic twins. Preventive screening of carriers with DICER1 mutations is important and follow-up is undertaken as recommended by the 2016 International PPB Register. CASE PRESENTATION: We present two pairs of monozygotic twins. In one pair of 4-year, 2-month old girls, both with DICER1 mutation, one developed PPB(II) and her identical sibling had acute transient hepatitis. In the other pair of 19-month-old female babies, one had a history of bronchopulmonary hypoplasia and developed PPB(III) without DICER1 mutation, and her identical sibling had allergic asthma. Both patients with PPB were treated with R0 resection and received 12 cycles of postoperative chemotherapy. At the most recent review, the twins had been followed up for six and eight years, respectively, and they all remained healthy. However, the height and weight of the patients with PPB were lower than those of their respective identical sister. CONCLUSIONS: PPB is rare, especially in monozygotic twins. We emphasise the importance of genetic testing and follow-up in monozygotic twins with PPB. During the follow-up, children surviving PPB should be monitored closely for growth and development disorders which caused by chemotherapy.

Economic burden for retinoblastoma patients in China.

BACKGROUND: Although retinoblastoma (Rb) is considered to have a good prognosis, economical stress is still a huge problem for patients' families. Besides, doctors, the government, and social foundation staff do not precisely know how much is truly required for complete Rb therapy, especially the non-medical costs and indirect costs. This study was conducted to estimate the economic burden of Rb patients. METHODS: This was a retrospective study. Fifty Rb patients were finally enrolled in the study. The questionnaire survey was conducted with surviving Rb patient' main family caregivers to collect the information on costs during illness through the phone. Costs included direct and indirect cost; direct costs included medical and non-medical costs. Medical costs include drug costs, surgery costs, treatment fees, lab tests, non-lab tests costs, and medical consumptive stuff costs (including hospital expenses and outpatient fees). RESULTS: The total direct cost was $27,814.62 ± 15,137.73, and the average medical cost was $15,034.48 ± 8,224.19 ($3,963.99-36,826.53). The total non-medical expenses averaged $12,252.93 ± 9,872.64 ($728.86-48,104.95). The average reduced working time was 11.50 ± 8.06 months, and the average lost income was $13,512.23 ± 11,545.83. Among the non-medical expenses, the average non-medical expenses for children in Beijing and surrounding areas was $6,557.68 ± 6,385.42, and the average non-medical expenses for children in other provinces and was $14,502.29 ± 10,484.86, t-test p-value = 0.011. The average transportation cost for children in Beijing and surrounding areas (Hebei, Tianjin) was $1,871.09 ± 1,428.91, other provinces was $4,909.62 ± 3,697.02. Of children in Beijing and surrounding areas the average accommodation fee was $2,788.42 ± 3,065.00, in other provinces it was $6,599.27 ± 3,065.00. CONCLUSION: Children with Rb have a heavy economic burden. Direct non-medical expenses are higher. Getting medical treatment nearby can help reduce the economic burden of the disease. Besides, work-related issues are also a major financial problem for families with Rb, and the government should properly provide economic subsidies. Simplifying the national health insurance process and purchasing commercial supplementary medical insurance will increase the family's ability to afford the cost of cancer treatment.

Analysis of treatment cost for neuroblastoma to the family: a single-center cross-sectional study in China.

BACKGROUND: Neuroblastoma (NB) is notorious in childhood cancer because of its high incidence and poor prognosis. The Children's Oncology Group reported that the 3-year OS in the high-risk (HR) group is 50%, and the HR-NB with bone marrow metastasis in our center is 43.1%. Thousands of families in China suffer from the cost of NB, but the true costs of therapy are unknown; to date, no study has ever performed a detailed therapy costs analysis for NB. The objective of this study was to assess the economic burden of NB treatment in children to the family, to ultimately reduce related expenses for patients and promote the establishment of NB management policy. MATERIALS AND METHODS: Data in this cross-sectional study were collected via questionnaires completed by parents at the outpatient clinic and was verified via a computer system. Therapy costs of children with NB of differing risks were analyzed through descriptive statistics (1 CNY ≈ 0.1412 USD). RESULTS: Median direct medical costs of low risk (LR), middle risk (MR), and HR NB during treatment were 180.0 (120.0, 300.0), 200.0 (166.0, 300.0), and 650.0 (415.5, 850.0) thousand Chinese yuan (CNY), respectively. Direct non-medical costs including transportation, food, and accommodation were 60.0 (37.0, 100.0), 80.0 (60.0, 120.0), and 100.0 (80.0, 157.5) thousand CNY in the LR, MR, and HR groups, respectively. Additionally, parents accrued work absences to attend treatment, and lost a total of 100.0 (50.0, 150.0) thousand CNY in indirect costs. LIMITATIONS: Families whose children had relapsed or died were excluded from this analysis and therefore limited the conclusions drawn. Parents were asked to recall costs since initial diagnosis (1-6 years in the past), but this extended time period may have introduced recall bias. CONCLUSIONS: Direct non-medical and indirect costs play an important role in the total treatment costs of NB. Children with NB treated in local hospitals and followed up in hospital specialized in childhood oncology may save many unnecessary expenses. China's healthcare system should establish mechanisms and provide financial support for children with NB.