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Background Cystic fibrosis related diabetes (CFRD) has been associated with pulmonary function decline, nutritional status decline and increased mortality. In 2010, the CFRD Clinical Care Guideline were updated, recommending all patients with CF begin CFRD screening at 10 years old. This study uses CF Foundation Patient Registry to examine the impact of screening practices at centers across the United States from 2008 to 2015. We examined the association of screening practices and CFRD diagnosis at individual centers and trends in ppFEV1 and BMI percentile prior to and after diagnosis. Methods The cohort was defined as patients with CF and without CFRD who turned 10 years old from 2009 to 2015. Centers were classified based on their CFRD screening rates. Kaplan-Meier curves summarized the distribution of age at CFRD diagnosis. Among patients diagnosed with CFRD, we examined differences in ppFEV1 and BMI percentile two years prior to diagnosis and two years post-diagnosis by clinic screening rate. Results Of 3553 patients, 445 (13%) were diagnosed with CFRD. The average age of diagnosis was 13 years old. The screening rate of the patients' clinic was significantly associated with time to diagnosis (pvalue=0.0001). Among patients diagnosed with CFRD, clinics with lower screening rates of CFRD had steeper rates of pulmonary decline two years prior to diagnosis. Conclusion Centers that screen for CFRD more tend to diagnose a larger percent of patients with CFRD and at a younger age. Additionally, patients at centers with lower screening rates have faster rates of pulmonary decline prior to CFRD diagnosis.

BACKGROUND: Localized prostate cancer (PCa) treatments provide high survival rates, with patients often surviving a decade or longer after treatment. Therefore, treatment options are progressively based on quality of life. The objective of this research was to investigate magnitude of response shift (RS) in health-related quality of life (HRQOL) responses in men with clinically localized PCa using a generic questionnaire and a disease-specific questionnaire in an observational longitudinal patient registry study. PATIENTS AND METHODS: A cohort study was conducted using the Cancer of the Prostate Strategic Urologic Research Endeavor (CaPSURE) database. Patients were annually surveyed using the Medical Outcomes Study Questionnaire Short Form 36 (SF-36) and the UCLA Prostate Cancer Index (PCI) HRQOL measures. A total of 3161 active patients were eligible for a one-off supplemental study asking retrospective HRQOL scores (then-test). We calculated RS, observed change, and RS adjusted change. Statistical difference was determined by t test. RESULTS: Patients consistently reported higher recalled pretreatment HRQOL compared to baseline scores for SF-36 and PCI, confirming the existence of a RS (P < .05). On average, PCI demonstrated larger RS by a factor of 2 than SF-36. More specific, RS was greater especially in SF-36 physical domains compared to mental health items. PCI measured PCa-specific physical adverse effects only. Patients whose cancer had recurred reported slightly lower SF-36 RS than those whose cancer had not recurred. CONCLUSION: RS occurrence was measured in both the disease-specific questionnaire and the generic HRQOL questionnaire, demonstrating continued low health and symptom scores after RS adjustment. Therefore, health professionals should adjust for this phenomenon when assessing patient's HRQOL treatment responses, and clinicians should address their continued sexual and urinary functional loss.

Lingyan You,1– 3 Hongtao Niu,1,2 Ke Huang,1,2 Fen Dong,1,2,4 Ting Yang,1,2 Chen Wang1,2,5 1Department of Pulmonary and Critical Care Medicine, Center of Respiratory Medicine, China-Japan Friendship Hospital, Beijing, People’s Republic of China; 2National Clinical Research Center for Respiratory Diseases, Beijing, People’s Republic of China; 3Department of Pulmonary and Critical Care Medicine, Quanzhou First Hospital Affiliated Fujian Medical University, Quanzhou, Fujian, People’s Republic of China; 4Institute of Clinical Medical Sciences, China-Japan Friendship Hospital, Beijing, People’s Republic of China; 5Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, People’s Republic of ChinaCorrespondence: Ting YangDepartment of Pulmonary and Critical Care Medicine, Center of Respiratory Medicine, China-Japan Friendship Hospital, No. 2, East Yinghua Road, Chaoyang District, Beijing, 100029, People’s Republic of ChinaEmail zryyyangting@163.comChen WangChinese Academy of Medical Sciences and Peking Union Medical College, 9 Dongdan 3rd Alley, Dong Dan, Dongcheng, Beijing, 100730, People’s Republic of ChinaEmail cyh-birm@263.netPurpose: To identify clinical features and outcomes associated with pulmonary heart disease among patients with chronic obstructive pulmonary disease exacerbation (COPD), which may help reduce economic burden accrued over hospital stay and shorten length of stay (LOS).Patients and Methods: Totally, 4386 patients with acute exacerbation of COPD (AECOPD) classified into pulmonary heart disease (PHD) group and non-pulmonary heart disease group, were included from the ACURE registry, a prospective multicenter patient registry study. Clinical features and outcomes were compared between groups.Results: PHD patients had a more severe profile, including having higher scores of COPD assessment test and modified British Medical Research Council, worse lung function, more patients hospitalized more than once in the past year due to acute exacerbation of COPD, and more comorbidities. Furthermore, drug cost was higher and length of stay was longer in AECOPD patients with PHD.Conclusion: AECOPD patients with PHD had a more severe profile and worse clinical outcomes, including higher drug cost and longer LOS. PHD is an independent risk factor of drug cost and LOS. Complicated with PHD in COPD/AECOPD patients with PHD means heavier disease burden and worse prognosis. It merits further study to focus on PHD management in COPD/AECOPD patients.Keywords: chronic obstructive pulmonary disease, exacerbation, pulmonary heart disease, cost, length of stay

Background Diabetes is a common comorbidity among patients with exacerbation of chronic obstructive pulmonary disease (AECOPD). Diabetes has been reported to be associated with length of stay (LOS), death, and cost among AECOPD patients. However, most studies are retrospective or have small sample sizes. The association for cost has not been researched using diabetes as a main analytic factor. This study aimed to fill gaps mentioned above, to compare basic characteristics between the diabetic and non-diabetic group, and to detect associations between diabetes and clinical outcomes among patients hospitalized with AECOPD. Methods A total of 5334 AECOPD patients, classified into diabetic and non-diabetic group, were included from a prospective multicenter patient registry study. Generalized linear regression and logistic regression were separately used for the association between diabetes and direct hospitalization cost and the association between diabetes and LOS. Results Generally, diabetic patients had a more severe profile, including being older, more overweight or obese, having more former smokers, more emergency room visits in the past 12 months, and more comorbidities occurrence. Diabetic patients also had worse clinical outcomes, including higher cost and longer LOS. Additionally, the generalized linear regression indicated that the marginal mean cost difference between diabetic and non-diabetic patients was RMB (¥) 775.7. Conclusions AECOPD patients with comorbid diabetes had a more severe profile and higher direct hospitalization cost. Diabetes screening and integrated care programs might help reduce the heavy comorbidity and economic burden. Moreover, corticosteroids and metformin could be considered in the treatment of these patients. Trial registration Clinicaltrials.gov with the identifier NCT0265752.

Premature ovarian insufficiency (POI) affects seriously physical and mental health of the patients. Studies show that acupuncture may be promising in treating POI, yet evidence is insufficient. International patient registry, one form of observational study, is designed for evaluating effectiveness and safety of acupuncture in real-world clinical settings. We will face a series of opportunities and challenges. The opportunities are as follows: (1) Interdisciplinary cooperation can improve the effect of acupuncture for POI. (2) The protocol for acupuncture to treat POI has room for improvement. (3) The patient registry study can make up the shortcomings of randomized controlled trials. (4) The patient registry study can collect real-world data and explore responses of different populations to acupuncture and can be the foundation for the successive studies. International patient registry may face technological, ethical and financial challenges, while the established International Patient Registry Platform of Acupuncture-Moxibustion ( www.amreg.org:8082/v3 ) is actively responding to these challenges. Till now there have been more than 300 patients of POI enrolled on this platform.

Aim To describe the total burden of disease in individuals with cerebral palsy (CP) in Norway. Method A comprehensive set of disorder categories were extracted from the Norwegian Patient Registry using International Statistical Classification of Diseases, 10th Revision diagnosis codes for individuals born between 1996 and 2010 who received specialist healthcare between 2008 and 2017 (0–21y). Individuals with CP were identified through a validation study in cooperation with the Cerebral Palsy Registry of Norway. Risk differences (proportions of individuals recorded with each disorder) were used to compare individuals with CP with the general population without CP. Results The study included 966 760 individuals. Among these, 2302 (0.24%) had CP (1330 males, 972 females). Of the individuals with CP, 95.0% were recorded with one or more comorbidity, and the risks of medical, neurological, and mental/behavioural disorders were higher compared with the risks in the general population. The most common neurological and mental/behavioural disorders were cocausal, i.e. attributed to the same injury to the developing brain that caused CP, while medical disorders were most often complications of CP or coincidentally co‐occurring with CP. Interpretation Individuals with CP have a considerably higher burden of medical, neurological, and mental/behavioural disorders compared with the general population, including disorders that are not directly caused by, or complications to, the brain injury. What this paper adds Nearly all individuals with cerebral palsy (CP) had one or more comorbidity. Fifty‐two per cent had at least one comorbidity attributed to the same cause as CP, complications of CP, and coincidentally co‐occurring with CP. Risks of medical, neurological, and mental/behavioural disorders were considerably higher than in the general population. © 2019 The Authors. Developmental Medicine & Child Neurology published by John Wiley & Sons Ltd on behalf of Mac Keith Press This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial‐NoDerivs License

Background Asthma is a heterogeneous disease with different phenotypes, endotypes and responses to treatment. Dust mite allergic asthma (DMAA) is the most common type in children. Compared with randomized control trials, a patient registry study (PRS) can reflect the real physical condition and clinical diagnosis more comprehensively. Methods Children who visited the asthma clinic of the Children's Hospital of Chongqing Medical University between August 2018 and August 2020, and met the inclusion criteria and also agreed to participate, were enrolled in the registry study. Clinical information, laboratory tests and peripheral blood samples were collected after informed consent was given by guardians. Results To date, 208 children have been enrolled in the patient registry database of DMAA. They are mainly male, with >50% having a history of allergic rhinitis, cesarean section, positive family history and passive smoking. Eosinophils and total immunoglobulin E levels were all significantly higher than normal. According to results for the childhood asthma control test (c-ACT) and ratio of forced expiratory volume in 1 s to predicted value after inhaled corticosteroid treatment, the uncontrolled group had higher hemoglobin (Hb) levels than the control group. The group exhibiting abnormal pulmonary function was older, and had longer disease duration, higher fractional exhaled nitric oxide and Hb than the group in which pulmonary function was restored. Conclusions We have preliminarily established a registered study database of children with DMAA. By cluster analysis and using blood samples, we can further study the different pathophysiological mechanisms in order to provide more individualized and targeted treatments for all children.

The Cystic Fibrosis Foundation Patient Registry (CFFPR) is an ongoing patient registry study that collects longitudinal demographic, clinical, and treatment information about persons with cystic fibrosis (CF) in the United States. CF is a life-shortening genetic disorder that occurs in approximately 1 in 3,500 births in the United States. High-quality observational data is important for clinical research, quality improvement, and clinical management.To describe the data collection, patient population, and key limitations of the CFFPR.Inclusion criteria for the CFFPR include diagnosis with CF or a CFTR-associated disorder, care at an accredited care center program, and provision of informed consent. Data from clinic visits and hospitalizations are collected through a secure website. Loss to follow-up and generalizability were examined using several methods. The accuracy of CFFPR data was evaluated with an audit of 2012 CFFPR data compared to the medical record.Since 1986, the CFFPR contains the records of 48,463 individuals with CF. Participation among individuals seen at accredited care centers is high, and loss to follow-up is low. An audit of 2012 CFFPR data suggests that the CFFPR contains 95% of clinic visits and 90% of hospitalizations found in the medical record for these patients, and nearly all of the audited fields were highly accurate.Registries such as the CFFPR are important tools for research, clinical care, and tracking incidence, mortality and population trends.

Introduction: Roflumilast (ROF) is approved by the European Medicines Agency for maintenance treatment of severe COPD associated with chronic bronchitis and a history of frequent exacerbations as add on to inhalation treatment. The aim of the Patient registry of ROFlumilast In real LifE (PROFILE) study was to capture real-world data on the use of ROF in standard clinical practice. Methods: PROFILE (ClinicalTrials.gov:NCT02015767) was an observational, non-interventional, non-comparative, prospective patient registry study. Patients initiating ROF were identified and followed for 1 year according to usual clinical practice. Data were collected at 3-monthly intervals. The primary outcome was the incidence and frequency of COPD exacerbations over 1 year. Results: 1473 patients were enrolled from 5 European countries (Bulgaria, Germany, Greece, Norway and Slovakia), of whom 1222 had valid records. Patients were of median age 67 years, predominantly male (71.4%), had severe/very severe airflow limitation (71.4%/10.5%) and experienced a median of 2 COPD exacerbations (5–95% percentile 0–8) in the prior year. The most common reasons for starting ROF were COPD progression (62.7%), high exacerbation rate (56.5%) and inadequate response to therapy (46.4%). During the 1-year follow-up, 214 (17.5%) patients had 316 COPD exacerbations (69 had >1 exacerbation); the median number of exacerbations was 0 (5–95% percentile 0–2). There were no significant differences in exacerbation rate between COPD phenotypes (emphysema, chronic bronchitis and combined phenotype). Conclusions: These results suggest that the use of ROF in standard clinical practice may reduce the frequency of COPD exacerbations.

CONTEXT: Epidemiological data for central diabetes insipidus (CDI) are sparse.OBJECTIVE: The purpose of this study was to provide accurate epidemiological data on CDI on a national level.DESIGN AND SETTING: This was a drug utilization and patient registry study during a 5-year period from 2007 to 2011.METHODS: We used the Danish National Prescription Registry data linked with the Danish National Patient Registry to study the epidemiology of CDI using waiting time distribution and other pharmacoepidemiological methods.PATIENTS: A total of 1285 patients with CDI were recorded in the observation period and given 9309 prescriptions for desmopressin in the nasal formulation, orodispersible tablet, or conventional tablet.RESULTS: The period prevalence rate of CDI in Denmark over the 5-year period investigated was 23 CDI patients per 100 000 inhabitants, with a higher prevalence in children and older adults (>80 years of age). The 1-year period prevalence rate of CDI decreased in Denmark over the 5 years from approximately 10 to 7 CDI patients per 100 000 inhabitants. The yearly incidence rate of new cases of CDI was found to be 3 to 4 patients per 100 000. The incidence of (presumable) congenital CDI was found to be 2 infants per 100 000 infants. Half of the patients with CDI prescribed as oral treatment were provided dosing instructions to only administer the drug before bedtime, and one third of the CDI patients either had no specific instructions or were instructed to use the drug as needed. Hospital admissions due to severe hyponatremia occurred in 0.9% of patients over a 5-year period, predominantly in females with an incidence ratio of women to men of 1.8:1.CONCLUSION: Half of the cases of CDI are acquired later in life. At least half of the patients with CDI are instructed to prevent nocturnal polyuria, but it is not clear whether their CDI remains uncontrolled during the daytime or, alternatively, whether they use desmopressin only as needed. Female patients with CDI had approximately twice the number of hospital admissions due to severe hyponatremia than male patients with CDI.

Background Thrombocytopenia is a common complication of critical illness and an independent risk factor for bleeding and death in the intensive care unit (ICU). Platelet transfusions are commonly used to improve platelet counts; however, the expected platelet increment from a transfusion in this setting has not been established. The objective of this study was to describe the frequency of platelet transfusion administration and their effect on platelet count increments in a large cohort of non-oncology critically ill adults. Methods We performed an analysis of a registry database, which was developed to capture clinical and laboratory data on all blood transfusions administered in 3 academic hospitals in Hamilton, Ontario, Canada. We included all patients ≥18 years who received one or more platelet transfusion during an ICU admission. Data validation was done by integrity checks with medical records and laboratory information system performed by a biostatistician. Non-transfused ICU patients were used as controls. The absolute increment in platelet count was calculated for each single platelet transfusion using the closest platelet count taken within 24 hours before the transfusion and 4-24 hours after the transfusion. Results Between April 2006 and October 2012, 33,222 patients were admitted to ICU, including 29,511 (88.8%) who did not have a diagnosis of cancer. Of those, 4,502 (15.3%) received one or more platelet transfusion during any ICU admission (n=4,690); 31.9% were female and median age at the time of first admission was 69 years (IQR 59-77). Among the 25,009 non-transfused patients admitted to ICU during the same period, 38.1% were female and the median age was 65 years (IQR 52–76). Median pre-transfusion platelet count was 87 x109/L (IQR 59-131) and a single platelet transfusion resulted in a median platelet count increment of 21 x109/L (IQR 6-40) as measured 6.7 hours (IQR 5.1-9.8) after the transfusion. There were 277 (25.4%) transfusions that yielded a platelet count increment of 5 x109/L or less. ICU mortality was 562/4,690 (12.4%) for patients who received a platelet transfusion, compared with 2,251/33,033(6.8%) for patients who were not transfused during their ICU stay. Summary/Conclusion Among this large cohort of non-oncology ICU patients, platelet transfusions were commonly administered for thrombocytopenia that was generally mild. In this setting one platelet transfusion resulted in a median platelet count rise of 21 x109/L. Many transfusion episodes yielded no appreciable increase in platelet count. Further studies are needed to determine the clinical effects of platelet transfusion in this setting controlling for confounding. Disclosures: Heddle: CIHR: Research Funding; Canadian Blood Services: Membership on an entity’s Board of Directors or advisory committees; Health Canada: Research Funding; Macopharma: Consultancy; ASH: Honoraria.