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Aim: To assess treatment patterns and outcomes in patients with non-del(5q) lower-risk myelodysplastic syndromes. Methods: Patient medical records were reviewed in the USA, Canada (CAN), UK and the EU. Results: Analysis included 119 patients in the USA/CAN (median age, 61.5 years) and 245 patients in the UK/EU (median age, 67.3 years). Most patients received erythropoiesis-stimulating agents (ESAs) as first-line (1L) therapy (USA/CAN: 89.0%; UK/EU: 90.2%). A substantial proportion of 1L erythropoiesis-stimulating agent-treated patients were transfusion dependent before 1L (USA/CAN: 37.1%; UK/EU: 51.2%); a small percentage of these patients achieved transfusion independence during 1L therapy (USA/CAN: 2.8%; UK/EU: 14.4%). Conclusion: These findings highlight an unmet need for more effective treatments among patients with non-del(5q) lower-risk myelodysplastic syndromes.
[Box: see text].
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Aim: To assess real-world ruxolitinib treatment patterns and outcomes in patients diagnosed with primary or secondary myelofibrosis. Materials & methods: Patient medical records were reviewed in six countries. Results: Eligible patients (n = 469) had a mean age of 63.5 years, and most were male (66.5%) with primary myelofibrosis (78.5%). Median duration of ruxolitinib treatment was 13.1 months; 40% of patients initiated treatment at the recommended dose. The Kaplan-Meier estimate of median survival from ruxolitinib initiation was 44.4 months (95% CI, 38.8-50.2 months). Approximately one quarter (23%) of patients continued ruxolitinib after progression. Conclusion: These results suggest an unmet need for more effective treatments for patients with myelofibrosis who failed ruxolitinib.
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Mielofibrose Primária , Feminino , Humanos , Masculino , Prontuários Médicos , Pessoa de Meia-Idade , Nitrilas/uso terapêutico , Mielofibrose Primária/tratamento farmacológico , Pirazóis/uso terapêutico , Pirimidinas/uso terapêutico , Resultado do TratamentoRESUMO
PURPOSE OF REVIEW: Immune checkpoint inhibitors (ICIs) have improved the survival of several cancers. However, they may cause a wide range of immune-related adverse events (irAEs). While most irAEs are manageable with temporary cessation of ICI and immunosuppression, cardiovascular toxicity can be associated with high rates of morbidity and mortality. As ICIs evolve to include high-risk patients with preexisting cardiovascular risk factors and disease, the risk and relevance of ICI-associated cardiotoxicity may be even higher. RECENT FINDINGS: Several cardiovascular toxicities such as myocarditis, stress cardiomyopathy, and pericardial disease have been reported in association with ICIs. Recent findings also suggest an increased risk of atherosclerosis with ICI use. ICI-associated myocarditis usually occurs early after initiation and can be fulminant. A high index of suspicion is required for timely diagnosis. Prompt treatment with high-dose corticosteroids is shown to improve outcomes. Although the overall incidence is rare, ICI cardiotoxicity, particularly myocarditis, is associated with significant morbidity and mortality, making it a major therapy-limiting adverse event. Early recognition and prompt treatment with the cessation of ICI therapy and initiation of high-dose corticosteroids are crucial to improve outcomes. Cardio-oncologists will need to play an important role not just in the management of acute cardiotoxicity but also to reduce the risk of long-term sequelae.
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Aterosclerose/diagnóstico , Cardiotoxicidade/diagnóstico , Inibidores de Checkpoint Imunológico/uso terapêutico , Miocardite/diagnóstico , Neoplasias/tratamento farmacológico , Aterosclerose/induzido quimicamente , Aterosclerose/imunologia , COVID-19/epidemiologia , COVID-19/prevenção & controle , COVID-19/virologia , Cardiotoxicidade/etiologia , Cardiotoxicidade/imunologia , Humanos , Inibidores de Checkpoint Imunológico/efeitos adversos , Inibidores de Checkpoint Imunológico/imunologia , Miocardite/induzido quimicamente , Miocardite/imunologia , Neoplasias/imunologia , Pandemias , Fatores de Risco , SARS-CoV-2/isolamento & purificação , SARS-CoV-2/fisiologiaRESUMO
OBJECTIVE: Assess patient characteristics, real-world treatment patterns, and health care resource utilization (HCRU) among patients with psoriatic arthritis (PsA) in Japan. METHODS: Patients diagnosed with PsA from April 2009 through July 2017 were identified from the Medical Data Vision database. Patient characteristics, treatment patterns, and HCRU were evaluated for these patients. RESULTS: A total of 639 patients met inclusion criteria and were included in the analysis for patients with a PsA diagnosis. Over 12 months following diagnosis, patients received oral NSAIDs (61.7%), conventional synthetic disease-modifying antirheumatic drugs (DMARDs) (55.1%), corticosteroids (35.1%), topical NSAIDs (34.0%), adalimumab (14.7%), infliximab (9.7%), secukinumab (5.0%), ustekinumab (4.5%), ixekizumab (1.6%), and golimumab (1.6%). A total of 227 (35.5%) patients initiated biologic DMARDs (bDMARDs) over the median 25.2 months of study follow-up. Compared with the overall group of patients diagnosed with PsA, patients who initiated bDMARDs had higher median total per-patient health care costs ($27,772 vs. $11,316), lower median per-patient hospitalization costs ($31,164 vs. $39,359), and fewer median hospital days per admission (8.0 vs. 12.0 days). CONCLUSION: This study presents knowledge of the current state of patient characteristics, treatment patterns, HCRU, and costs among patients with PsA in Japan. Considering the relatively recent guideline recommendations, the preliminary treatment patterns suggest physicians may be following treatment guidelines.
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Antirreumáticos , Artrite Psoriásica , Adalimumab/uso terapêutico , Antirreumáticos/uso terapêutico , Artrite Psoriásica/diagnóstico , Artrite Psoriásica/tratamento farmacológico , Hospitais , Humanos , Japão , Estudos RetrospectivosRESUMO
OBJECTIVES: To understand the current state of treatment patterns and health care resource utilization among patients in Japan with ankylosing spondylitis (AS) managed in the real-world setting. METHODS: Patient records from the Medical Data Vision database were analyzed to identify patients with ICD-10 AS from April 2009 through July 2017. Measures evaluated included demographic, clinical, and other characteristics at diagnosis; treatment patterns; health care resource utilization; and costs. RESULTS: Four hundred and seventeen patients met the study's inclusion criteria. Treatments observed during the first year after the initial AS diagnosis included nonsteroidal anti-inflammatory drugs (79.6%), corticosteroids (39.3%), methotrexate (22.3%), sulfasalazine (16.8%), adalimumab (14.2%), and infliximab (12.2%). At any time during the mean 33 months of study follow-up, biologic disease-modifying antirheumatic drugs (bDMARDs) were initiated by 115 patients. During the study follow-up, patients who initiated bDMARDs had higher median total per-patient annual health care costs ($26,937 vs $15,323), lower median per-patient hospitalization costs ($29,817 vs. $39,509), and fewer median hospital days per admission (7.0 vs. 11.0 days) compared with the overall group of patients diagnosed with AS. CONCLUSION: This database study provides knowledge of patient characteristics, treatment patterns, HCRU, and costs for patients with AS in Japan. The study outcomes demonstrate a need for increased awareness of proper AS management.
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Utilização de Instalações e Serviços/estatística & dados numéricos , Custos de Cuidados de Saúde/estatística & dados numéricos , Espondilite Anquilosante/tratamento farmacológico , Adalimumab/uso terapêutico , Adulto , Antirreumáticos/uso terapêutico , Feminino , Hospitais/estatística & dados numéricos , Humanos , Infliximab/uso terapêutico , Japão , Masculino , Metotrexato/uso terapêutico , Pessoa de Meia-Idade , Espondilite Anquilosante/economiaRESUMO
BACKGROUND: Cancer and cardiovascular disease (CVD) are independently associated with adverse outcomes in patients with COVID-19. However, outcomes in patients with COVID-19 with both cancer and comorbid CVD are unknown. METHODS: This retrospective study included 2,476 patients who tested positive for SARS-CoV-2 at 4 Massachusetts hospitals between March 11 and May 21, 2020. Patients were stratified by a history of either cancer (n=195) or CVD (n=414) and subsequently by the presence of both cancer and CVD (n=82). We compared outcomes between patients with and without cancer and patients with both cancer and CVD compared with patients with either condition alone. The primary endpoint was COVID-19-associated severe disease, defined as a composite of the need for mechanical ventilation, shock, or death. Secondary endpoints included death, shock, need for mechanical ventilation, need for supplemental oxygen, arrhythmia, venous thromboembolism, encephalopathy, abnormal troponin level, and length of stay. RESULTS: Multivariable analysis identified cancer as an independent predictor of COVID-19-associated severe disease among all infected patients. Patients with cancer were more likely to develop COVID-19-associated severe disease than were those without cancer (hazard ratio [HR], 2.02; 95% CI, 1.53-2.68; P<.001). Furthermore, patients with both cancer and CVD had a higher likelihood of COVID-19-associated severe disease compared with those with either cancer (HR, 1.86; 95% CI, 1.11-3.10; P=.02) or CVD (HR, 1.79; 95% CI, 1.21-2.66; P=.004) alone. Patients died more frequently if they had both cancer and CVD compared with either cancer (35% vs 17%; P=.004) or CVD (35% vs 21%; P=.009) alone. Arrhythmias and encephalopathy were also more frequent in patients with both cancer and CVD compared with those with cancer alone. CONCLUSIONS: Patients with a history of both cancer and CVD are at significantly higher risk of experiencing COVID-19-associated adverse outcomes. Aggressive public health measures are needed to mitigate the risks of COVID-19 infection in this vulnerable patient population.
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OBJECTIVE: The objective of this article is to describe real-world treatment patterns and outcomes in patients with platinum-refractory/resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer (PRROC) in the United States, United Kingdom, and Canada. METHODS/MATERIALS: Physicians retrospectively reviewed medical records of women aged 18 years or older who were diagnosed with PRROC between January 2010 and June 2014. Patient characteristics, initial PRROC therapy, and health care utilization were assessed; progression-free survival (PFS) and overall survival were estimated using Kaplan-Meier and Cox proportional hazards methods. RESULTS: Data were obtained on 392 US, 296 UK, and 82 Canadian patients. At initial ovarian cancer diagnosis, 65.8% (United States), 93.3% (United Kingdom), and 82.9% (Canada) of patients had stage III/IV disease; 43.6%, 73.7%, and 56.1%, respectively, had high-grade tumors. At PRROC diagnosis, mean age was 57.2 years (United States), 59.2 years (United Kingdom), and 57.4 years (Canada). Eastern Cooperative Oncology Group performance status was 0/1 at PRROC diagnosis for 57.7% (United States), 80.1% (United Kingdom), and 36.6% (Canada) of patients. Most patients initiated systemic treatment after PRROC diagnosis (United States, 71.4%; United Kingdom, 83.1%; Canada, 81.7%). The most common initial PRROC therapy was pegylated liposomal doxorubicin monotherapy (United States, 18.6%; United Kingdom, 50.0%; Canada, 34.3%). During initial PRROC treatment, 80.7%, 59.8%, and 44.8% of patients had 1 office visit or more and 17.5%, 10.2%, and 14.9% of patients had 1 hospitalization or more in the United States, the United Kingdom, and Canada, respectively. Treatment toxicity was the most common reason for hospitalization (United States, 75.5%; United Kingdom, 64.0%; Canada, 80.0%). Median (95% confidence interval) PFS was 5.6 (4.9-6.2), 8.0 (6.8-9.2), and 6.4 (5.4-9.3) months in the United States, the United Kingdom, and Canada. The Cox proportional hazards model showed that stage III/IV, high-grade tumors, and poorer performance status were associated with shorter survival. CONCLUSIONS: Current treatments for PRROC yield limited PFS and frequent hospitalizations reported to be related to toxicities or procedural complications, suggesting a continued unmet need for more effective and tolerable therapeutic strategies for PRROC.
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Antineoplásicos/uso terapêutico , Carboplatina/uso terapêutico , Carcinoma Epitelial do Ovário/tratamento farmacológico , Resistencia a Medicamentos Antineoplásicos , Neoplasias Ovarianas/tratamento farmacológico , Idoso , Carcinoma Epitelial do Ovário/mortalidade , Feminino , Humanos , Pessoa de Meia-Idade , Neoplasias Ovarianas/mortalidade , Estudos Retrospectivos , Resultado do TratamentoRESUMO
After an arterial switch operation (ASO), serial imaging is necessary to monitor for maladaptive changes. We compared cardiac magnetic resonance imaging (CMR) to 2-D transthoracic echocardiography (TTE) in assessing post-operative ASO patients. We performed a retrospective review of patients at a single tertiary care center who underwent an ASO and subsequently had a CMR performed from 7/2010 to 7/2016. Those with single ventricle anatomy, congenitally corrected transposition of the great arteries, or previous atrial switch operation were excluded. TTE obtained within 6 months of the CMR was used for comparison. Parameters compared included ventricular size and systolic function, semilunar valve regurgitation, neo-aortic root dimension, and the presence of branch pulmonary artery (PA) stenosis (on CMR by the Nakata index or right/left flow differential; on TTE by peak velocity > 2 m/s or PA diameter Z score < - 2). Forty-seven patients with 90 CMR and 86 TTE studies met inclusion criteria. CMR and TTE assessment of right ventricular (RV) and left ventricular function did not statistically differ. RV dilation was overdetected by TTE (p = 0.046). Right pulmonary artery and left pulmonary artery (LPA) visualization by TTE was worse than CMR (p < 0.01). There was no statistically significant difference between CMR and TTE assessment of branch PA stenosis; however, there was poor agreement between the use of Z score and velocity when determining branch PA stenosis by TTE (κ < 0). Assessment of neo-pulmonary regurgitation (PR) and neo-aortic regurgitation (AR) was significantly different between CMR and TTE (p < 0.05). Assessment for delayed enhancement was performed in 18% of CMR studies (n = 16), with perfusion defects appreciated in three patients. Substantial differences between CMR and TTE exist when examining the post-operative ASO patient. CMR was superior for evaluation of the branch PAs, which commonly require re-intervention. TTE failed to recognize altered ventricular function in several cases. Differences between TTE and CMR could alter management is some cases. Incorporation of CMR into the routine surveillance of patients who received an ASO is warranted.
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Transposição das Grandes Artérias/métodos , Ecocardiografia/métodos , Imagem Cinética por Ressonância Magnética/métodos , Adolescente , Aorta/diagnóstico por imagem , Transposição das Grandes Artérias/efeitos adversos , Criança , Pré-Escolar , Feminino , Valvas Cardíacas/diagnóstico por imagem , Ventrículos do Coração/diagnóstico por imagem , Ventrículos do Coração/fisiopatologia , Humanos , Recém-Nascido , Masculino , Monitorização Fisiológica/métodos , Período Pós-Operatório , Artéria Pulmonar/diagnóstico por imagem , Estudos Retrospectivos , Transposição dos Grandes Vasos/cirurgiaRESUMO
BACKGROUND: Mycobacterium goodii is an infrequent human pathogen which has been implicated in prosthesis related infections and penetrating injuries. It is often initially misidentified as a gram-positive rod by clinical microbiologic laboratories and should be considered in the differential diagnosis. CASE PRESENTATION: We describe here the second reported case of M. goodii endocarditis. Species level identification was performed by 16S rDNA (ribosomal deoxyribonucleic acid) gene sequencing. The patient was successfully treated with mitral valve replacement and a prolonged combination of ciprofloxacin and trimethoprim/sulfamethoxazole. CONCLUSION: Confirmation of the diagnosis utilizing molecular techniques and drug susceptibility testing allowed for successful treatment of this prosthetic infection.
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Endocardite Bacteriana/diagnóstico , Endocardite Bacteriana/patologia , Anuloplastia da Valva Mitral/efeitos adversos , Infecções por Mycobacterium não Tuberculosas/diagnóstico , Infecções por Mycobacterium não Tuberculosas/patologia , Mycobacterium/classificação , Mycobacterium/isolamento & purificação , Idoso , Antibacterianos/uso terapêutico , Análise por Conglomerados , DNA Bacteriano/química , DNA Bacteriano/genética , DNA Ribossômico/química , DNA Ribossômico/genética , Ecocardiografia Transesofagiana , Endocardite Bacteriana/tratamento farmacológico , Endocardite Bacteriana/cirurgia , Coração/diagnóstico por imagem , Humanos , Masculino , Mycobacterium/genética , Infecções por Mycobacterium não Tuberculosas/tratamento farmacológico , Infecções por Mycobacterium não Tuberculosas/cirurgia , Filogenia , RNA Ribossômico 16S/genética , Radiografia Torácica , Reimplante , Análise de Sequência de DNA , Tomografia Computadorizada por Raios X , Resultado do TratamentoRESUMO
Hydatid cyst is a tissue parasitic infection caused by tapeworm Echinococcus granulosus. Common location for hydatid cysts are the liver (65%) and the lungs (25%). Cardiac hydatid cyst is seen rarely, occurring in about 0.5-2% of all cases of hydatid disease. We present this case of 45 years female who presented with short duration of dry cough and atypical chest pain. Chest X ray showed cardiomegaly with round bulge at the right heart border and curvilinear calcification in left upper abdomen in the region of spleen. Transthoracic echocardiography (TTE) depicted cystic lesion in Right Ventricle free wall causing compression of right atrial and ventricular cavity. Cardiac CT confirmed this cyst as hydatid cyst. Patient underwent successful excision of right ventricular hydatid cyst.
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Equinococose/complicações , Equinococose/diagnóstico por imagem , Cardiopatias/diagnóstico por imagem , Cardiopatias/parasitologia , Doenças Assintomáticas , Equinococose/cirurgia , Ecocardiografia , Feminino , Cardiopatias/cirurgia , Humanos , Pessoa de Meia-Idade , Radiografia Torácica , Tomografia Computadorizada por Raios XRESUMO
Iron deficiency anemia is a significant problem worldwide and more so in developing countries, like India. The prevention and treatment of iron deficiency is a major public health goal in India It is now well recognized that iron deficiency has detrimental effects in patients with coronary artery disease, heart failure, and pulmonary hypertension, and possibly in patients undergoing cardiac surgery. Around one-third of all patients with HF, and around one-half of patients with pulmonary hypertension, are affected by iron deficiency.1.
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Anemia Ferropriva , Compostos Férricos/farmacologia , Insuficiência Cardíaca , Anemia Ferropriva/complicações , Anemia Ferropriva/diagnóstico , Anemia Ferropriva/tratamento farmacológico , Insuficiência Cardíaca/complicações , Insuficiência Cardíaca/diagnóstico , Insuficiência Cardíaca/terapia , Hematínicos/farmacologia , Humanos , Administração dos Cuidados ao Paciente/métodos , Resultado do TratamentoRESUMO
Pulmonary thromboembolism is common and missed by clinicians. We report a case of massive pulmonary embolism which was life threatening treated by the catheter assisted technique. Anticoagulation is the mainstay of therapy for most patients, with thrombolytic therapy reserved for some patients.1 Recent studies have suggested a role for systemic or catheter-directed thrombolytic therapy in selected patients.2 We present a case of a patient who presented with an PE, was successfully treated with catheter-directed thrombolysis.
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Fibrinolíticos , Embolia Pulmonar , Terapia Trombolítica , Catéteres , Fibrinolíticos/administração & dosagem , Humanos , Embolia Pulmonar/terapia , Terapia Trombolítica/métodos , Resultado do TratamentoAssuntos
Cardiomiopatias/tratamento farmacológico , Terapia Baseada em Transplante de Células e Tecidos/métodos , Linfoma não Hodgkin/tratamento farmacológico , Receptores de Antígenos Quiméricos/uso terapêutico , Adulto , Idoso , Idoso de 80 Anos ou mais , Humanos , Pessoa de Meia-Idade , Estudos Retrospectivos , Adulto JovemRESUMO
BACKGROUND: Previous economic evaluations compared specific chemotherapy agents using input parameters from clinical trials and resource utilization costs. Cost-effectiveness of treatment groups (drug classes) using community-level effectiveness and cost data, however, has not been assessed for elderly patients with breast cancer. OBJECTIVE: To assess the cost-effectiveness of chemotherapy regimens by age and disease stage under "real-world" conditions for patients with breast cancer. METHODS: The Surveillance Epidemiology and End Results-Medicare data were used to identify patients with breast cancer with American Joint Committee on Cancer stage I/II/IIIa, hormone receptor-negative (estrogen receptor-negative and progesterone receptor-negative) patients from 1992 to 2009. Patients were categorized into three adjuvant treatment groups: 1) no chemotherapy, 2) anthracycline, and 3) non-anthracycline-based chemotherapy. Median life-years and quality-adjusted life-years (QALYs) were measured using Kaplan-Meier analysis and were evaluated against average total health care costs (2013 US dollars). RESULTS: A total of 4575 patients (propensity score-matched) were included for the primary analysis. The anthracycline group experienced 12.05 QALYs and mean total health care costs of $119,055, resulting in an incremental cost-effectiveness ratio of $7,688 per QALY gained as compared with the no chemotherapy group (QALYs 7.81; average health care cost $86,383). The non-anthracycline-based group was dominated by the anthracycline group with lower QALYs (9.56) and higher health care costs ($122,791). Base-case results were found to be consistent with the best-case and worst-case scenarios for utility assignments. Incremental cost-effectiveness ratios varied by age group (range $3,790-$90,405 per QALY gained). CONCLUSIONS: Anthracycline-based chemotherapy was found cost-effective for elderly patients with early stage (stage I, II, IIIa) breast cancer considering the US threshold of $100,000 per QALY. Further research may be needed to characterize differential effects across age groups.
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Antraciclinas/economia , Antraciclinas/uso terapêutico , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Neoplasias da Mama/tratamento farmacológico , Anos de Vida Ajustados por Qualidade de Vida , Fatores Etários , Idoso , Idoso de 80 Anos ou mais , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Análise Custo-Benefício , Feminino , Humanos , Estimativa de Kaplan-Meier , Medicare/estatística & dados numéricos , Estadiamento de Neoplasias , Programa de SEER , Fatores Socioeconômicos , Estados UnidosRESUMO
OBJECTIVES: Most economic evaluations of chemotherapies for ovarian cancer patients have used hypothetical cohorts or randomized control trials, but evidence integrating real-world survival, cost, and utility data is limited. METHODS: A propensity score-matched cohort of 6856 elderly (≥65 years) ovarian cancer patients diagnosed from 1991 to 2005 from the Surveillance, Epidemiology, and End Results-Medicare data cohort were included. Treatment regimens (i.e., no chemotherapy, platinum-based only, platinum plus taxane, and other nonplatinum) were identified in the 6 months postdiagnosis. Patients were followed until death or end of study (December 2006). Effectiveness was measured in quality-adjusted life-years (QALYs), and total health care costs were measured by using a payer's perspective (2009 US dollars). Methodological and statistical uncertainties were accounted by including alternative scenarios (for utility values) and net monetary benefit approach. Incremental cost-effectiveness ratios (ICERs) were calculated, and stratified analyses were performed by tumor stages and age groups. RESULTS: On comparing the platinum-based group versus no chemotherapy, we found that the ICER was $30,073/QALY and $58,151/QALY for early- and late-stage disease, respectively, while other nonplatinum and platinum plus taxane groups were dominated (less effective and more costly). Similar results were found across alternative scenarios and age groups. For patients 85 years or older, platinum plus taxane, however, was not dominated by the platinum-based group, with an ICER of $133,892/QALY. CONCLUSIONS: Following elderly ovarian cancer patients over a lifetime using real-world longitudinal data and adjusting for quality of life, we found that treatment with platinum-based regimen was the most cost-effective treatment alternative.
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Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Medicare/economia , Neoplasias Ovarianas/tratamento farmacológico , Compostos de Platina/economia , Compostos de Platina/uso terapêutico , Taxoides/economia , Taxoides/uso terapêutico , Idoso , Idoso de 80 Anos ou mais , Análise Custo-Benefício , Feminino , Custos de Cuidados de Saúde , Humanos , Modelos Econômicos , Estadiamento de Neoplasias , Neoplasias Ovarianas/patologia , Pontuação de Propensão , Anos de Vida Ajustados por Qualidade de Vida , Programa de SEER , Taxa de Sobrevida , Resultado do Tratamento , Estados UnidosRESUMO
Background and Aims: Limited real-world nontertiary care evidence on the patient therapeutic journey and disease burden of eosinophilic esophagitis (EoE) exists. The aim was to collect real-world data on the EoE patient journey across different age groups. Methods: This retrospective, real-world, cohort study used electronic medical records and claims data provided by a rural integrated US healthcare system. Eligibility criteria included ≥ 2 diagnoses of EoE (2009-2018), ≥ 1 endoscopy, and ≥ 12 months of data before and after the index date (the first endoscopy date during the 180 days before and the 365 days after the first EoE diagnosis). Clinical findings, all-cause healthcare resource utilization, specialists consulted, therapies, and markers of disease progression were analyzed. Results: Overall, 613 patients were enrolled: 0-11 (children, n = 182), 12-17 (adolescents, n = 146), 18-54 (adults, n = 244), and ≥ 55 years old (older adults, n = 41). Post index, the prevalence of signs and symptoms increased. At baseline, most endoscopies were abnormal (80.5%) and most peak eosinophil counts were > 15 eosinophils/high-power field (87.9%); post index, all age groups had endoscopic and histologic improvements. However, 3 years post index, abnormal endoscopic appearance (62.3%) and histologic activity (51.2%) were observed. Patients of all ages exhibited considerable all-cause healthcare resource utilization. During follow-up, 86.3% of patients consulted a specialist. Before and after index, proton pump inhibitors and corticosteroids were the most commonly used pharmacological therapies; 44.0% of patients discontinued their first treatment post index. Disease progression occurred in 13.9% of patients post index. Conclusion: In this setting, patients with EoE irrespective of age face difficult therapeutic journeys with substantial disease burden.
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Aim: To examine clinical characteristics, real-world treatment patterns, and health outcomes among patients with germline BRCA1/2-mutated, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer (ABC). Methods: A retrospective analysis was conducted using medical records from patients with HER2-negative ABC with BRCA1/2 mutation who received cytotoxic chemotherapy. Data were stratified into groups with triple-negative breast cancer (TNBC) or hormone receptor-positive (HR+)/HER2-negative diagnoses. Time-to-event outcomes (i.e., real-world progression-free survival [rwPFS] and overall survival [OS]) were calculated to summarize health outcomes. Results: When diagnosed with ABC, most patients were younger than 60 years (mean age = 57.3 years), were white (76.4%), and had a family history of BRCA-related cancer (71.5%). A total of 305 patient records were examined; 194 patients (63.6%) had advanced TNBC, and 111 patients (36.4%) had HR+/HER2-negative ABC. Chemotherapy was primarily used as first-line treatment for both subgroups, but the TNBC subgroup received poly (ADP-ribose) polymerase (PARP) inhibitors at triple the rate as a second-line treatment and double the rate as a third-line treatment compared with the HR+/HER2-negative subgroup. Two-year OS rates were similar between the TNBC (73.9%) and the HR+/HER2-negative subgroups (77.0%), and anemia, nausea, and neutropenia were the most commonly reported toxicities across all treatments. Conclusion: Clinicians should consider the use of targeted agents such as PARP inhibitors in earlier lines of therapy for ABC given the growing evidence that PARP inhibitors may improve PFS compared with chemotherapy while potentially offering a more manageable toxicity profile and improved quality of life.
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Distal renal tubular acidosis (dRTA), also known as Type 1 renal tubular acidosis, is a rare disorder. It primarily occurs through the inability to secrete H+ ions. The causes of dRTA can be divided into primary and secondary. The most common secondary cause of dRTA is Sjögren syndrome. dRTA typically presents as hypokalemia with non-anion gap metabolic acidosis. Here, we present a patient where Sjögren's syndrome causing dRTA was masked by the presence of hypernatremia causing metabolic acidosis with a high anion gap.
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Acidose Tubular Renal , Hipernatremia , Síndrome de Sjogren , Humanos , Síndrome de Sjogren/complicações , Síndrome de Sjogren/diagnóstico , Acidose Tubular Renal/diagnóstico , Acidose Tubular Renal/etiologia , Acidose Tubular Renal/complicações , Feminino , Hipernatremia/etiologia , Hipernatremia/diagnóstico , Equilíbrio Ácido-Base , Pessoa de Meia-IdadeRESUMO
Aim: To describe real-world testing patterns for RET in US patients with advanced/metastatic medullary thyroid cancer and determine consistency of real-world testing practices with national guidelines. Materials & methods: The authors performed a retrospective medical record analysis of patients with advanced/metastatic medullary thyroid cancer who initiated systemic therapy between 2013 and 2018. Seventy-five US-based oncologists collected the data using a customized electronic data collection form. Results: A total of 59.6% (121 of 203) of patients underwent testing for RET, and 37.2% (45 of 121) had a RET mutation, of which 55.6% were identified as RET mutation-positive before initial diagnosis. Overall, 90 (44.3%) patients were tested for biomarkers on or after initial diagnosis, with RET being the most tested (95.6%) biomarker. Conclusion: The authors' findings suggest an opportunity to improve testing rates in accordance with treatment guidelines.
Mutations in the RET gene are common in patients with medullary thyroid cancer (MTC). As RET mutations are involved in the development of MTC, several treatment guidelines recommend counseling patients and testing for mutations in the RET gene in all patients with MTC. However, limited data are available on RET testing patterns in the USA in this patient population. In this study, the authors determined testing patterns for RET in patients with advanced or metastatic MTC in the USA using real-world data and found that only 60% of patients were tested for RET (i.e., testing for presence of RET mutations was observed in less than two-thirds of all patients included in the study). These results demonstrate the need for improved testing for RET mutations in patients with MTC in alignment with the treatment guidelines in routine clinical practice in the USA.