Cancers develop resistance to inhibitors of oncogenes mainly due to target-centric mechanisms such as mutations and splicing. While inhibitors or antagonists force targets to unnatural conformation contributing to protein instability and resistance, activating tumor suppressors may maintain the protein in an agonistic conformation to elicit sustainable growth inhibition. Due to the lack of tumor suppressor agonists, this hypothesis and the mechanisms underlying resistance are not understood.
View Article and Find Full Text PDFSeveral studies reported that patients with acute myeloid leukemia (AML) who remain in long-term remission after allogeneic or autologous transplant have a shorter life expectancy, compared to the general population. However, little is known about the life expectancy of adult long-term survivors of AML who were treated with chemotherapy alone without a transplant and there have been no comparisons with survival among the general population. The current study indicates that the life expectancy of AML patients who achieved and maintained CR for at least 3 years is shorter than expected for age in the US population.
View Article and Find Full Text PDFBackground: Cardiovascular disease is the leading cause of death among patients with Duchenne muscular dystrophy (DMD). Identifying patients at risk of early death could allow for increased monitoring and more intensive therapy. Measures that associate with death could serve as surrogate outcomes in clinical trials.
View Article and Find Full Text PDFWe present a rare case of pan-valvular involvement in a 5-month-old female with Kawasaki disease shock syndrome despite early treatment with intravenous immunoglobulin and corticosteroids. She experienced a favorable outcome after the addition of infliximab, which was guided based on clinical, laboratory and echocardiogram findings, rather than recrudescence of fever, the most common indicator of intravenous immunoglobulin resistance.
View Article and Find Full Text PDFBackground: Obesity (body mass index [BMI] ≥30 kg/m ) is an important epidemiological risk factor for developing acute myeloid leukemia (AML). Therefore, the authors studied the association of obesity with clinical and genetic phenotype and its impact on outcome in adults with AML.
Methods: The authors analyzed BMI in 1088 adults who were receiving intensive remission induction and consolidation therapy in two prospective, randomized therapeutic clinical trials of the Eastern Cooperative Oncology Group-American College of Radiology Imaging Network: E1900 (ClinicalTrials.
Purpose: Patients with hematologic malignancies are more optimistic than their oncologists and their expectations may be a barrier to timely hospice care. Patient expectations toward the end of life (EOL), however, have not been characterized. In this study, we analyzed interviews of bereaved caregivers to understand the expectations of patients diagnosed with acute myeloid leukemia and the factors that influenced those expectations, from diagnosis until death.
View Article and Find Full Text PDFPurpose: Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) experience in specific MDs and in limited numbers. This study sought to describe the outcomes associated with VAD therapy in an unselected population across multiple centers.
View Article and Find Full Text PDFThe life-limiting complications of Duchenne muscular dystrophy (DMD) include loss of lung function and progressive cardiomyopathy; when patients are treated with assisted ventilation, cardiac function becomes the main determinant of survival. Therapy for DMD is changing rapidly, with the emergence of new genetic and molecular therapeutic options, the proliferation of which has fostered the perception that DMD is a potentially curable disease. However, data for respiratory and cardiac outcomes are scarce and available evidence is not uniformly positive.
View Article and Find Full Text PDFCardiac disease has emerged as a leading cause of mortality in Duchenne muscular dystrophy in the current era. This survey sought to identify the diagnostic and therapeutic approach to DMD among pediatric cardiologists in Advanced Cardiac Therapies Improving Outcomes Network. Pediatric cardiology providers within ACTION (a multi-center pediatric heart failure learning network) were surveyed regarding their approaches to cardiac care in DMD.
View Article and Find Full Text PDFDystrophin deficiency results in cardiomyopathy and fibrosis with variable onset. Little is known about electrocardiographic abnormalities in Becker muscular dystrophy and their relationship to underlying cardiac pathology. We hypothesized QRS fragmentation is associated with myocardial fibrosis on cardiac magnetic resonance imaging in Becker muscular dystrophy patients.
View Article and Find Full Text PDFContext: Goals-of-care communication (GOCC) is recommended to increase the value of cancer care near the end of life (EOL).
Objectives: Conduct a systematic review of the evidence that GOCC is associated with higher-value care.
Methods: We searched PubMed, Scopus, Ovid MEDLINE, EMBASE, EMB Reviews, CINAHL, and PsycInfo from inception to July 2019.
There are conflicting reports in the literature suggesting that one gender or the other has a better survival with acute myeloid leukaemia (AML). The present study was done in an attempt to resolve the issue. The effect of gender was examined on 3546 newly diagnosed patients with AML, including 548 patients with acute promyelocytic leukaemia (APL) enrolled in 10 multi-institutional treatment studies from March 1984 to November 2008.
View Article and Find Full Text PDFAims: The inability to prescribe blood transfusions is a potential barrier to timely hospice enrollment for patients with blood cancers. The benefits and harms of transfusions near the end of life (EOL), however, are poorly characterized and patients' preferences are unknown. We sought to characterize the recollections of bereaved caregivers about the relationships between transfusions and hospice enrollment decisions.
View Article and Find Full Text PDFCardiac disease is now the leading cause of death in Duchenne muscular dystrophy (DMD). Clinical evaluations over time have demonstrated asymptomatic cardiac troponin elevations and acute elevations are associated with symptoms and cardiac dysfunction in DMD. Clinicians require a better understanding of the relationship of symptoms, troponin levels and progression of cardiac disease in DMD.
View Article and Find Full Text PDFA 17-year-old male patient who was diagnosed with Becker muscular dystrophy (nonsense mutation [c.3822C>A] within exon 28 of the DMD gene) at 6 years of age was evaluated in the multidisciplinary neuromuscular clinic for loss of ambulation for 1 year. From a pulmonary perspective, there were no acute or chronic respiratory symptoms, and no history of pneumonia or aspiration.
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