Annual prevalence estimation of lymphatic malformation with a cutaneous component: observational study of a national representative sample of physicians.

BACKGROUND: Lymphatic malformations (LMs) represent a potentially life-threatening, rare disease of the lymphatic system characterized by development of abnormal vessels, outpouchings, or cysts filled with lymphatic fluid. There are three morphologic types of LMs based on the size of the individual cysts: macrocystic (typically > 2 cm), microcystic (generally < 2 cm), and mixed (includes aspects of both). Macrocystic LMs typically exist beneath the skin and often can involve vascular components and/or organs. Microcystic LMs often have a cutaneous component and clinically present with lymphorrhea, bleeding, pain, itching, malodor, and functional deficits. There are no treatments approved by the US Food and Drug Administration (FDA) for either macrocystic or microcystic lymphatic malformations. The totality of the epidemiologic literature for LM is limited to the incidence of the disease among various birth cohorts. This is the first nationally representative study to estimate the national managed prevalence for patients with microcystic LM or combined LM with a cutaneous component annually across physician specialties likely to manage this condition. We conducted a retrospective observational survey of a nationally representative sample of patient-care physicians in the United States most likely to manage lymphatic malformations with a cutaneous component (LMC). Once recruited, target physicians participated via an electronic questionnaire. We weighted study physician self-estimates of the number of LMC patients treated in the past 12 months to reflect the specialists' corresponding proportion in the national universe. All patient information was anonymous; no personally identifiable information was collected. RESULTS: Of the 420 physicians who visited the study website, 316 agreed to be screened and to participate (75.2% participation rate). Our survey results indicated the estimated number of unique annually managed LMC patients by target specialists is 79,920 (CI 66,600-93,250). This number corresponds to managed prevalence of 24.1 LMC patients per 100,000 population (CI 19.6/100,000-28.4/100,000). CONCLUSIONS: The study indicates that while rare, LMC affects a substantial number of people in the US (79,920) who are being managed by one or more specialists. By better understanding the prevalence of people living with LMC who require treatment, efforts to both increase disease awareness and to identify underserved populations in need of potential new treatments can be better focused.

Epidemiology of nontuberculous mycobacterial pulmonary disease in Europe and Japan by Delphi estimation.

BACKGROUND: Nontuberculous mycobacterial pulmonary disease (NTM-PD) is an emerging opportunistic infection, but basic epidemiological data are lacking in most regions. We have investigated epidemiology and diagnostic and treatment practices in five EU countries (United Kingdom, Spain, Italy, France, Germany; EU5) and Japan. STUDY DESIGN: and methods: Annual prevalence in each country was established using a 2-round Delphi method in combination with a regional prevalence-estimation model that incorporated data obtained from a blinded physician screening survey (3154 physicians) and a real-world NTM-PD treating-physician/patient-chart observational study (619 physicians - 1429 patient charts). RESULTS: The annual prevalence of NTM-PD was estimated at 6.2/100,000 in the EU5 and 24.9/100,000 in Japan. Overall prevalence between the EU5 was comparable, while differences in regional prevalence were found to be pronounced in France and The United Kingdom. Regional differences were also found in Japan, with the majority of cases in Chubu and Kanto regions. CONCLUSION: This new methodology for obtaining often missing regional-level epidemiological data reveals dramatic variations in NTM-PD annual prevalence and helps pinpoint areas that may merit special preventative and treatment focus.

Adult Presentations of Congenital Midgut Malrotation: A Systematic Review.

BACKGROUND: Adult midgut malrotation is a rare cause of an acute abdomen requiring urgent intervention. It may also present in the non-acute setting with chronic, non-specific symptoms. The objective of this study is to identify the clinical features, appropriate investigations and current surgical management associated with adult malrotation. METHODS: A systematic review was conducted according to PRISMA guidelines, identifying confirmed cases of adult malrotation. Patient demographics, clinical features, investigation findings and operative details were analysed. RESULTS: Forty-five reports met the inclusion criteria, totalling 194 cases. Mean age was 38.9 years (n = 92), and 52.3% were male (n = 130). The commonest presenting complaints were abdominal pain (76.8%), vomiting (35.1%) and food intolerance (21.6%). At least one chronic symptom was reported in 87.6% and included intermittent abdominal pain (41.2%), vomiting (12.4%) and obstipation (11.9%). Computerised tomography scanning was the most frequent imaging modality (81.4%), with a sensitivity of 97.5%. The whirlpool sign was observed in 30.9%; abnormalities of the superior mesenteric axis were the commonest finding (58.0%). Ladd's procedure was the most common surgical intervention (74.5%). There was no significant difference in resolution rates between emergency and elective procedures (p = 0.46), but length of stay was significantly shorter for elective cases. (p = 0.009). There was no significant difference in risk of mortality, or symptom resolution, between operative and conservative management (p = 0.14 and p = 0.44, respectively). CONCLUSION: Malrotation in the adult manifests with chronic symptoms and should be considered as a differential diagnosis in patients with abdominal pain, vomiting and food intolerance.

Real-world adverse events with niraparib 200 mg/day maintenance therapy in ovarian cancer: a retrospective study.

Aim: To assess real-world occurrence of common clinical trial-reported adverse events (AE) among patients with recurrent ovarian cancer initiating niraparib 200 mg/day. Materials & methods: This retrospective observational study used physician-extracted anonymized medical record data of eligible patients initiating niraparib 200 mg/day after platinum-based chemotherapy. Results: Of 153 patients, 57 (37%) experienced ≥1 of the three most common all-grade AEs within 3 months after niraparib initiation: nausea (16%; grade 3/4: 2%), thrombocytopenia (14%; grade 3/4: 3%) and fatigue (24%; grade 3/4: 3%). In the ENGOT-OV16/NOVA trial, these respective AEs occurred in 74, 61 and 59% of patients. Conclusion: Incidence of common clinical trial-reported AEs was lower among patients initiating niraparib 200 mg/day in real-world practice versus patients initiating niraparib 300 mg/day in ENGOT-OV16/NOVA.

Repository corticotropin injection in patients with advanced symptomatic sarcoidosis: retrospective analysis of medical records.

BACKGROUND: Repository corticotropin injection (RCI) has regulatory approval for many indications, including symptomatic sarcoidosis. This large case series of patients with advanced symptomatic sarcoidosis treated with RCI describes patient characteristics, RCI utilization patterns, concomitant therapies, and physicians' assessments of treatment response. METHODS: Patients ⩾18 years with symptomatic sarcoidosis, treated with RCI in the previous 36 months, who had completed a course of RCI or received RCI for ⩾6 months at the time of data collection were included. RESULTS: The study included 302 patients (mean age, 51 years; 52%, women) with a mean 4.8 years since initial diagnosis of sarcoidosis. Most patients (76%) had extrapulmonary involvement, primarily in the skin (28%), joints (25%), heart (22%), and eyes (22%); 34% had multiple (⩾2) organ involvement. The mean duration of RCI treatment was 32.5 weeks, with 61.6% of patients continuing RCI therapy for ⩾6 months. The RCI utilization pattern indicated an individualized approach to therapy, with a higher starting dose associated with a shorter duration of therapy compared with a lower starting dose. The percentage of patients who used corticosteroids decreased from 61.3% during the 3 months before initiation of RCI to 12.9% 3 months after RCI therapy; the mean daily dose of corticosteroid decreased from 18.2 mg to 9.9 mg. The proportion of patients given <10 mg/day of prednisone increased from 21% before RCI use to 47% 3 months after RCI use. According to physicians' assessments of change in patients' health status after RCI therapy, overall status improved in 95% of patients, overall symptoms in 73%, lung function in 38%, and inflammation in 33%. CONCLUSIONS: The findings suggest that RCI is a viable treatment option for patients with advanced symptomatic sarcoidosis and provide insights on patient characteristics and practice patterns to help clinicians determine appropriate use. The reviews of this paper are available via the supplemental material section.

Retrospective Medical Record Review to Describe Use of Repository Corticotropin Injection Among Patients with Uveitis in the United States.

INTRODUCTION: Repository corticotropin injection (RCI) has immune-modulatory and anti-inflammatory effects and is approved for multiple indications, including severe and acute chronic allergic and inflammatory processes involving the eye and adnexa. This study describes patient characteristics, treatment patterns, and physicians' assessments of patients with uveitis treated with RCI. METHODS: This was a retrospective medical record review of US patients. Eligible patients had a diagnosis of uveitis, received RCI in the past 12 months, and had completed or were receiving RCI treatment at the time of data collection. Baseline characteristics and after-treatment clinical data are descriptively reported. RESULTS: The study included 91 patients (mean age 41 years, 62% female, and mean time since diagnosis 3.98 years). Most patients had moderate (n = 48, 53%) to severe (n = 21, 23%) visual impairment, and none was blind before RCI therapy. Patients used an average of 2.5 medications before RCI. Initial RCI dosing regimens, dose adjustments, and treatment durations were different for each patient. Concomitant medication use and dosages were reduced during RCI; 76 patients (84%) improved, 15 patients (16%) stayed the same, and none worsened; 86% of patients had improvements in vision. CONCLUSIONS: Physicians individualized RCI therapy among patients who suffered uveitis for several years and when previous therapies were inadequate. Most patients improved after initiating RCI, most commonly in vision. The findings support use of RCI for uveitis and provide a better understanding of patient characteristics and practice patterns to guide appropriate use.

Understanding if, How, and Why Women with Prior Spontaneous Preterm Births are Treated with Progestogens: A National Survey of Obstetrician Practice Patterns.

Objective In 2017, the Society for Maternal-Fetal Medicine (SMFM) Guideline Committee reaffirmed that 17 α -hydroxyprogesterone caproate (17-OHPC) to prevent preterm birth (PTB) is underutilized. We sought to determine what drove progestogen treatment choice of obstetricians managing pregnant women with histories of 1+ singleton spontaneous PTBs (< 37 weeks) who then delivered singleton gestations within the previous 12 months. Subjects We recruited a nationally representative random sample of obstetricians to abstract medical records of study-qualified patients. Of the 423 study-qualified physicians contacted, 358 (85%) participated; 56 (16%) maternal fetal medicine specialists and 302 (84%) general obstetrician/gynecologists (OB/GYNs) extracted data from 991 eligible patient charts. Results Almost three-fourths of patients (73.6%) were treated with 17-OHPC; 18.6% received vaginal progesterone, and 11.8% were not treated. Key drivers of physicians' choice to (1) prescribe branded 17-OHPC were "FDA (Food and Drug Administration) approval" (52% relative influence [RI]) and "SMFM guidelines" (24% RI); (2) prescribe vaginal progesterone were "ease of administration" (32% RI) and "shortened cervix" (16% RI); and (3) not provide prophylaxis were "patient not informed of risk" (35% RI) and "no shortened cervix" (29% RI). Conclusion Study findings support SMFM's contention of continued 17-OHPC underutilization to prevent PTB. Need for additional physician education merits assessment along with appropriate follow-up actions.

A Retrospective Medical Record Review of Utilization Patterns and Medical Resource Use Associated with Repository Corticotropin Injection among Patients with Rheumatologic Diseases in the United States.

INTRODUCTION: Repository corticotropin injection (RCI) has anti-inflammatory and immune-modulatory effects and is approved for multiple indications, including several rheumatologic conditions. The aims of this nationally representative, retrospective, observational study were to describe patient characteristics, RCI treatment patterns, and barriers to RCI use in patients with rheumatologic disease, and to compare medical resource use (MRU) before and after RCI therapy. METHODS: A random sample of US physicians was recruited to abstract the medical records of deidentified patients with a diagnosis of rheumatoid arthritis (RA), psoriatic arthritis (PsA), dermatomyositis/polymyositis (DM/PM), or systemic lupus erythematosus (SLE) who had been treated with RCI in the previous 24 months. Patient characteristics and patterns of RCI use were identified. Mean MRU in the 3 months before and after RCI therapy was compared using paired-samples t tests. RESULTS: A total of 449 physicians abstracted the medical records of 217 RA, 190 PsA, 254 DM/PM, and 95 SLE patients. In all groups combined, patients had received a mean of 3.3 treatment medications before initiating RCI. Most patients (75%-94%) were receiving RCI for the first time, indicating that repeated courses of RCI were uncommon. RCI was used as bridge therapy in 18% of patients. Approximately 24% of patients encountered an obstacle in accessing RCI, primarily insurance-related. After RCI therapy, the number of hospitalizations and hospital days were significantly reduced for all cohorts (all P < 0.05), and the number of outpatient visits was significantly lower for all cohorts (P < 0.05) except the SLE cohort (P = 0.3230). Study limitations include potentially incomplete data in the medical records and a relatively short duration for capturing MRU changes. CONCLUSIONS: RCI was used primarily as late-line therapy in patients with rheumatologic diseases. Medical resource use was significantly lower in the 3 months after therapy compared with 3 months prior. This finding suggests that RCI may improve disease control and warrants further evaluation. FUNDING: Mallinckrodt Pharmaceuticals.

Utility of the Oncotype DX® Prostate Cancer Assay in Clinical Practice for Treatment Selection in Men Newly Diagnosed with Prostate Cancer: A Retrospective Chart Review Analysis.

INTRODUCTION: The 17-gene Oncotype DX® prostate cancer assay (Genomic Health Inc., Redwood City, California) is a validated, biopsy based gene expression assay that reports the Genomic Prostate Score. Combined with clinical risk features, Genomic Prostate Score provides an individualized estimation of disease aggressiveness at diagnosis. With this retrospective chart review we assessed the impact of incorporating the Oncotype DX Genomic Prostate Score on treatment recommendations and decisions for men with newly diagnosed low risk prostate cancer in community urology practices. METHODS: A total of 24 urologists who ordered the Oncotype DX prostate cancer assay soon after launch (May 2013) were invited to participate in the study. Clinicopathological data, Genomic Prostate Score results and treatment related information were retrieved from medical records. Data also were collected for a pre-Genomic Prostate Score baseline group diagnosed from May 2012 to April 2013. Descriptive analyses were performed to evaluate the proportion of men for whom active surveillance was recommended and used before and after the availability of Genomic Prostate Score. RESULTS: Overall 15 physicians contributing 211 patients (Genomic Prostate Score group 124, baseline group 87) participated in the chart review. Patients in the Genomic Prostate Score and baseline groups had comparable risk based on traditional clinical pathological features, with 82% with NCCN® very low or low risk disease. With Genomic Prostate Score the relative increase in active surveillance recommended was 22% (baseline 50% and Genomic Prostate Score 61%, absolute increase of 11%) and the relative increase in use of active surveillance was 56% (baseline 43% and Genomic Prostate Score 67%, absolute increase of 24%). Treatment recommendations for active surveillance were directionally consistent with assay reported risk. CONCLUSIONS: Genomic Prostate Score testing was associated with greater physician recommendation of and use of active surveillance in community clinical practices.

The use of immediate postoperative instillations of intravesical chemotherapy after TURBT of NMIBC among European countries.

PURPOSE: The aim of this study was to assess the use of immediate postoperative instillation of intravesical chemotherapy (IPOIC) after transurethral resection of bladder tumour (TURBT) of nonmuscle invasive bladder cancer (NMIBC) in Europe. METHODS: Urologists based in five European Union nations were asked to extract information from the records of patients with NMIBC-urothelial carcinoma who received at least one TURBT. Multivariate logistic regression models were developed to determine the significant predictors of IPOIC usage. Data were weighted to control for country-to-country and other differences. RESULTS: Overall, 324 urologists (58 France, 72 Germany, 62 Italy, 65 Spain, 67 United Kingdom) were involved; the participation rate was 55 %. Overall, 771 patients received 954 TURBTs (mean-1.2/patient), of which 413 of the TURBTs (43.3 %) were administered IPOIC . Sixty-six of the 413 IPOICs (16.0 %) were for a recurrent tumour. Five of the tested variables were significantly associated with a patient's likelihood of receiving IPOIC after TURBT. Variables in the order of significance are as follows: (1) country (United Kingdom, patients most likely to receive IPOIC; France, least likely); (2) progression risk (physician assessed) [lower-risk conditions (no CIS, tumour < 3 cm) or intermediate risk-more likely]; (3) whether urologist completed a uro-oncology fellowship (completed-more likely); (4) recurrence risk (physician assessed) [higher-risk conditions (≥T2, ≥3 cm, CIS)-more likely]; and (5) physician's NMIBC patients volume (higher volume-more likely). CONCLUSIONS: This study revealed wide practice variation and substantial noncompliance with European Association of Urology Guidelines on the use of IPOIC after TURBT for NMIBC.

Lack of adherence to evidence-based treatment guidelines for nontuberculous mycobacterial lung disease.

RATIONALE: The 2007 American Thoracic Society (ATS) and Infectious Diseases Society of America (IDSA) recommend that patients with pulmonary nontuberculous mycobacterial (PNTM) disease caused by Mycobacterium avium complex (MAC) or M. abscessus be treated with a macrolide-based multidrug antibiotic regimen until sputum culture negative for 1 year. After 6 years, the degree of adherence to recommended guidelines among physicians remains unknown. OBJECTIVE: To describe antibiotic treatment practices among physicians treating patients with PNTM in the United States. METHODS: A nationally representative sample of 1,286 U.S. physicians was contacted in December 2011 through January 2012; 582 of the responding physicians were treating patients with PNTM and were eligible to participate. Physicians were asked to extract medical record data on the last four patients they treated in the past year with PNTM disease from either MAC or M. abscessus. Treatment patterns were assessed for all patients by NTM species and physician specialty, and compared with the 2007 recommended ATS/IDSA guidelines. MAIN RESULTS: Questionnaires were completed by 349 physicians on 915 patients with PNTM, including 744 (81%) with MAC and 174 (19%) with M. abscessus; 3 patients were positive for both. Physicians treated 76 (44%) patients with M. abscessus and 411 (55%) patients with MAC. Only 13% of antibiotic regimens prescribed to patients with MAC met ATS/IDSA guidelines, 56% did not include a macrolide, and 16% were for macrolide monotherapy. Among patients with M. abscessus, 64% of regimens prescribed did not include a macrolide. CONCLUSIONS: Adherence to the 2007 ATS/IDSA guidelines for treating PNTM disease is poor. Across all physician specialties evaluated, suboptimal or potentially harmful antibiotic regimens were commonly prescribed.

National practice patterns for immediate postoperative instillation of chemotherapy in nonmuscle invasive bladder cancer.

PURPOSE: We assessed the use of intravesical postoperative chemotherapy among United States urologists in patients with nonmuscle invasive bladder cancer. MATERIALS AND METHODS: A national sample of United States based urologists (259) retrospectively assessed their practice patterns involving intravesical postoperative chemotherapy after transurethral resection in patients with nonmuscle invasive bladder cancer. These urologists reviewed the medical records of their last 4 patients with nonmuscle invasive bladder cancer, and completed a case report form for specific demographic, pathological and treatment information. Selection criteria included the pathological and patient factors of histologically confirmed diagnosis of nonmuscle invasive bladder cancer-transitional cell carcinoma, completion of initial treatment plan with ongoing observation, candidate for or recipient of intravesical therapy, and no ongoing initial intravesical induction therapy. RESULTS: Overall the participation rate among those sampled was 61%. Of the 1,010 eligible patients with nonmuscle invasive bladder cancer 59.6% received instillation therapy during the initial treatment, of whom 28.4% (16.9% of patients overall) received intravesical postoperative chemotherapy. Primary, low risk patients most often received intravesical postoperative chemotherapy and 90.4% of the time patients received immediate instillation within 12 hours of surgery. However, of the urologists surveyed 66% never used intravesical postoperative chemotherapy, 17% used intravesical postoperative chemotherapy half (50%) of the time and only 2% used intravesical postoperative chemotherapy all (100%) of the time. CONCLUSIONS: Wide variation in the use of intravesical postoperative chemotherapy exists among urologists in the United States. The reason for the great diversity in the use of intravesical postoperative chemotherapy is speculative. However, physician awareness, physician bias, recurrence risk, and local pharmacy and hospital practice factors are all likely contributing factors.

Predictors of treatment choices and associated outcomes in actinic keratoses: results from a national physician survey study.

OBJECTIVES: Actinic keratoses (AKs) are common skin lesions with the potential to progress to squamous cell carcinoma. Many effective treatment alternatives exist, yet the reasons for treatment choice have not been explored. This study examined which AK therapy was preferred among dermatologists and primary care physicians (PCPs), as well as potential determinants of therapeutic selection. METHODS: A random national sample of 534 dermatologists and PCPs selected from the American Medical Association database completed AK questionnaires. The final sample included 1184 AK patients treated by dermatologists and 559 AK patients treated and/or referred by PCPs. All analyses were weighted using the survey sampling weights. RESULTS: Patients who had new and recurring lesions as well as patients who had a mean duration of more than a year since the last AK episode treatment (all p<0.05) were more likely to receive pharmacotherapy. Patients being treated by a dermatologist, receiving pharmacotherapy treatment only, and having both new and recurring lesions (all p<0.05) were less likely to have a complete treatment success. CONCLUSIONS: This study identifies several patient and physician factors associated with treatment preference and related outcomes in patients being treated for AK.

Treatment of biologically inactive nonunions by a limited en bloc ostectomy and compression plate fixation: a review of 17 cases.

OBJECTIVE: To describe a surgical technique for treatment of biologically inactive nonunions using en bloc ostectomy and compression plate fixation and clinical outcome in 17 dogs. STUDY DESIGN: Retrospective clinical study. SAMPLE POPULATION: Seventeen dogs. METHODS: A transverse ostectomy was performed adjacent and parallel to the nonunion to eliminate nonviable tissue and provide a new, viable fracture surface with a minimum circumferential contact area of 315 degrees. With most of the bony column anatomically reconstructed, compression plate fixation was used to stabilize the fracture. Autogenous cancellous bone grafting was used if a fracture gap was present (<45 degrees of missing circumferential bone contact). Resection of bone was limited so that bone shortening was less than 20% of the overall bone length. Clinical and radiographic follow-up evaluations were obtained whenever possible. RESULTS: Complete circumferential bone contact and compression plate fixation was achieved after ostectomy in 12 dogs; cancellous bone graft was used in 5 dogs. En bloc ostectomy sites were radiographically healed in a median time of 2.5 months after surgery in 11 dogs that returned for complete in-hospital follow-up, and progressive healing was observed in 3 other dogs, where in-hospital follow-up was obtained up to 2 months after surgery. These dogs had a median follow-up time of 2 months, at which time 6 dogs had no lameness, 4 had minimal lameness, and one had moderate lameness. No complications occurred, and no implants were removed. CONCLUSIONS: En bloc ostectomy with compression plate fixation was considered successful for the treatment of biologically inactive nonunions. A good to excellent prognosis can be expected with minimal complications. CLINICAL RELEVANCE: Use of an en bloc ostectomy technique for the treatment of biologically inactive nonunions permits easy resection of nonviable tissue. Subsequent fracture stabilization with compression plate fixation resulted in rapid bone healing without complications.