Robyn M. Emanuel

Bio: Robyn M. Emanuel is an academic researcher from Mayo Clinic. The author has contributed to research in topics: Quality of life & Myeloproliferative neoplasm. The author has an hindex of 7, co-authored 16 publications receiving 481 citations. Previous affiliations of Robyn M. Emanuel include Rosalind Franklin University of Medicine and Science.

Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: Prospective International Assessment of an Abbreviated Symptom Burden Scoring System Among Patients With MPNs

Abstract: Purpose Myeloproliferative neoplasm (MPN) symptoms are troublesome to patients, and alleviation of this burden represents a paramount treatment objective in the development of MPN-directed therapies. We aimed to assess the utility of an abbreviated symptom score for the most pertinent and representative MPN symptoms for subsequent serial use in assessing response to therapy. Patients and Methods The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (MPN-SAF TSS) was calculated as the mean score for 10 items from two previously validated scoring systems. Questions focus on fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers. Results MPN-SAF TSS was calculable for 1,408 of 1,433 patients with MPNs who had a mean score of 21.2 (standard deviation [SD], 16.3). MPN-SAF TSS results significantly differed among MPN disease subtypes (P < .001), with a mean of 18.7 (SD, 15.3), 21.8 (SD, 16.3), and 25.3 (SD, 17.2) f...

Cognitive Enhancement Drug Use Among Future Physicians: Findings from a Multi-Institutional Census of Medical Students

Abstract: BACKGROUND Nonmedical use of prescription psychostimulants such as methylphenidate and amphetamine salts for the purpose of cognitive enhancement is a growing trend, particularly in educational environments. To our knowledge, no recent studies have evaluated the use of these psychostimulants in a medical academic setting.

Patients with polycythemia vera have worst impairment of quality of life among patients with newly diagnosed myeloproliferative neoplasms.

Abstract: The quality of life (QoL) at the time of diagnosis of myeloproliferative neoplasm (MPN) has, to date, not been studied. One hundred and seventy-nine patients with MPN: 80 with essential thrombocythemia (ET), 73 with polycythemia vera (PV), 22 with primary myelofibrosis (PMF) and four with MPN undifferentiated, were included in this study. European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQC30) and the MPN-Symptom Assessment Form (MPN-SAF) were used to evaluate QoL. Fatigue was the most reported symptom in these patients. Patients with PV reported significantly higher mean scores for inactivity, dizziness, cough, itching, depression and lower total QoL compared to patients with ET. Patients with PV had significantly more headache and itching compared to patients with PMF. When the newly diagnosed patients with MPN were compared with a cohort of patients with MPN with mean disease duration of 7.8 years, the differences were most striking for patients with PMF, with significantly more fatigue, abdominal discomfort, concentration problems, insomnia, fever, weight loss and lower overall QoL developed over time.

Ruxolitinib in clinical practice for therapy of myelofibrosis: single USA center experience following Food and Drug Administration approval.

Abstract: Myelofi brosis is a myeloproliferative neoplasm (MPN) characterized by increased bone marrow fi brosis, abnormal blood counts with peripheral cytopenias, extramedullary hematopoiesis, splenomegaly and profound constitutional symptoms [1,2]. Presenting as a primary disease or arising from polycythemia vera or essential thrombocythemia, myelofi brosis incurs a signifi cant symptom burden for patients, and results in a reduced survival of 2 – 11 years [3 – 7]. Amongst MPNs, myelofi brosis has represented a unique challenge to eff orts aimed at palliating symptoms or impacting disease course. Th e 2005 discovery of the gain-of-function JAK2V617F mutation present in a signifi cant proportion of patients with polycythemia vera, essential thrombocythemia and myelofi brosis opened a new avenue of investigation into potential novel therapies [8,9]. Among the various JAK2 inhibitor agents that have been developed, ruxolitinib was the fi rst to receive Food and Drug Administration (FDA) approval in the USA for the treatment of intermediate- and high-risk myelofi brosis. Th e Controlled Myelofi brosis Study with Oral JAK Inhibitor Treatment Trials compared ruxolitinib therapy in patients with intermediate-2 or high-risk myelofi brosis with placebo (COMFORT-I) and with best available therapy (COMFORT-II) [10,11]. In both trials, patients in the ruxolitinib group experienced signifi cant clinical benefi ts, including reduction in spleen size and amelioration of debilitating symptoms coupled with a tolerable side-eff ect profi le. In this study, we report the initial clinical experience with ruxolitinib outside of the clinical trial setting at our high-volume MPN program. Approval for the analysis was obtained from the Mayo Clinic Institutional Review Board. Patients with myelofi brosis including primary myelofi brosis (PMF), post-polycythemia vera (post-PV MF) and post-essential thrombocythemia (post-ET MF) were included. All patients had a Dynamic International Prognostic Scoring System (DIPSS) score of intermediate-1/2 risk or high risk at the start of therapy [12]. Clinical and hematopathological data, including bone marrow biopsy, were reviewed in all cases, and the diagnosis was made in accordance with the 2008 World Health Organization criteria. Baseline patient information, physical examination results, laboratory data, hematopathology reports and interim follow-up information were acquired via chart review, with baseline obtained within 1 month of initiating therapy. All patients were prescribed ruxolitinib within 6 months of FDA approval. Ruxolitinib was started at 20 mg twice daily for patients with platelet counts of 200 10 9 /L or hemoglobin concentrations 10 g/dL. Patients with values below these were started on lower doses at the discretion of the providing physician. Doses were adjusted for lack of effi cacy or excess toxicity. Complete blood counts were checked on a weekly basis for the fi rst 2 months, then monthly thereafter. Drug effi cacy and toxicity were determined at follow-up visits and through telephone conversations with patients. Symptoms were assessed through a verbal response during review of systems questioning. Spleen size was determined by physical examination. Descriptive statistics were determined using means, medians, ranges and standard deviations for continuous variables. Categorical variables were evaluated using frequencies and relative frequencies.

Ruxolitinib versus Standard Therapy for the Treatment of Polycythemia Vera

Abstract: Background Ruxolitinib, a Janus kinase (JAK) 1 and 2 inhibitor, was shown to have a clinical benefit in patients with polycythemia vera in a phase 2 study. We conducted a phase 3 open-label study to evaluate the efficacy and safety of ruxolitinib versus standard therapy in patients with polycythemia vera who had an inadequate response to or had unacceptable side effects from hydroxyurea. Methods We randomly assigned phlebotomy-dependent patients with splenomegaly, in a 1:1 ratio, to receive ruxolitinib (110 patients) or standard therapy (112 patients). The primary end point was both hematocrit control through week 32 and at least a 35% reduction in spleen volume at week 32, as assessed by means of imaging. Results The primary end point was achieved in 21% of the patients in the ruxolitinib group versus 1% of those in the standard-therapy group (P<0.001). Hematocrit control was achieved in 60% of patients receiving ruxolitinib and 20% of those receiving standard therapy; 38% and 1% of patients in the two g...

Nestin-expressing progenitor cells: function, identity and therapeutic implications.

Abstract: The neuroepithelial stem cell protein, or Nestin, is a cytoskeletal intermediate filament initially characterized in neural stem cells. However, current extensive evidence obtained in in vivo models and humans shows presence of Nestin+ cells with progenitor and/or regulatory functions in a number of additional tissues, remarkably bone marrow. This review presents the current knowledge on the role of Nestin in essential stem cell functions, including self-renewal/proliferation, differentiation and migration, in the context of the cytoskeleton. We further discuss the available in vivo models for the study of Nestin+ cells and their progeny, their function and elusive nature in nervous system and bone marrow, and their potential mechanistic role and promising therapeutic value in preclinical models of disease. Future improved in vivo models and detection methods will allow to determine the true essence of Nestin+ cells and confirm their potential application as therapeutic target in a range of diseases.

A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase III trials of ruxolitinib for the treatment of myelofibrosis

Abstract: Ruxolitinib, a potent Janus kinase 1/2 inhibitor, resulted in rapid and durable improvements in splenomegaly and disease-related symptoms in the 2 phase III COMFORT studies. In addition, ruxolitinib was associated with prolonged survival compared with placebo (COMFORT-I) and best available therapy (COMFORT-II). We present a pooled analysis of overall survival in the COMFORT studies using an intent-to-treat analysis and an analysis correcting for crossover in the control arms. Overall, 301 patients received ruxolitinib (COMFORT-I, n=155; COMFORT-II, n=146) and 227 patients received placebo (n=154) or best available therapy (n=73). After a median three years of follow up, intent-to-treat analysis showed that patients who received ruxolitinib had prolonged survival compared with patients who received placebo or best available therapy [hazard ratio=0.65; 95% confidence interval (95%CI): 0.46–0.90; P=0.01]; the crossover-corrected hazard ratio was 0.29 (95%CI: 0.13–0.63). Both patients with intermediate-2– or high-risk disease showed prolonged survival, and patients with high-risk disease in the ruxolitinib group had survival similar to that of patients with intermediate-2–risk disease in the control group. The Kaplan-Meier estimate of overall survival at week 144 was 78% in the ruxolitinib arm, 61% in the intent-to-treat control arm, and 31% in the crossover-adjusted control arm. While larger spleen size at baseline was prognostic for shortened survival, reductions in spleen size with ruxolitinib treatment correlated with longer survival. These findings are consistent with previous reports and support that ruxolitinib offers a survival benefit for patients with myelofibrosis compared with conventional therapies. (clinicaltrials.gov identifiers: COMFORT-I, NCT00952289; COMFORT-II, NCT00934544)