scholarly journals 0766 Safety And Tolerability Of Pitolisant In The Treatment Of Adult Patients With Narcolepsy: Final Analysis Of An Open-label, Expanded Access Program In The United States

SLEEP ◽  
2020 ◽  
Vol 43 (Supplement_1) ◽  
pp. A291-A291
Author(s):  
E D Bauer ◽  
C W Davis ◽  
A Patroneva ◽  
J M Dayno ◽  
M J Thorpy
Keyword(s):  
United States ◽  
Development Program ◽  
Final Analysis ◽  
The United States ◽  
Adult Patients ◽  
Tolerability Profile ◽  
Open Label ◽  
Access To Treatment ◽  
Expanded Access ◽  
The Us

Abstract Introduction Pitolisant Expanded Access Clinical Evaluation (PEACE) provided adult patients with narcolepsy access to treatment with pitolisant while it was an investigational medication in the United States. Methods Pitolisant was titrated to 35.6 mg/day (or the highest tolerable dose) over a 3-week period. Dose adjustments were permitted at the discretion of the treating physician based on patient response. Treating physicians followed their standard of care and were required to report adverse events (AEs). Demographic and baseline information for all enrolled patients, and safety results available through October 30, 2019, are reported here (presentation will include final data from the PEACE program). Results In all, 623 patients (67.9% female; 84.6% white; mean age, 40.0 years; narcolepsy type 1, 51.5%) were treated with pitolisant in the PEACE program. Nearly all patients (98.4%) had been previously treated with other narcolepsy medications (88.1% with ≥2 narcolepsy medications). Overall, 35.2% of patients discontinued from the program; 16.7% due to an AE and 12.2% for lack of effect. At Month 1, 97.3% of patients remained in the study, 88.2% at Month 3, 76.5% at Month 6, 66.9% at Month 9, and 55.0% at Month 12. In all, 256 (41.1%) patients experienced ≥1 AE; majority (52.5%) of these AEs occurred early in treatment (by Week 3). The most commonly reported AEs were headache (9.8% of AEs), nausea (6.6%), anxiety (5.6%), and insomnia (4.7%). Conclusion In the PEACE program, patient characteristics were generally reflective of the US narcolepsy patient population. The safety and tolerability profile of pitolisant was similar to that seen in the clinical development program, with no new safety signals identified. The program ceased enrollment in August 2019 after the US approval of pitolisant for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. Support Harmony Biosciences, LLC.

scholarly journals Sun Protection Beliefs among Hispanics in the US

2014 ◽  
Vol 2014 ◽  
pp. 1-10 ◽  
Author(s):  
Marimer Santiago-Rivas ◽  
Chang Wang ◽  
Lina Jandorf
Keyword(s):  
United States ◽  
Skin Cancer ◽  
Risk Behaviors ◽  
Final Analysis ◽  
The United States ◽  
Sun Protection ◽  
Insufficient Evidence ◽  
Inclusion Criteria ◽  
Search Terms ◽  
The Us

Purpose. We reviewed the literature on sun protection beliefs in Hispanics living in the United States to explore what challenges are faced by area of research.Method. A review of PubMED, PsycINFO, and CINAHL databases was performed. Studies were published in peer-reviewed journals (in all years available) and written in English. The search terms used were [“skin cancer” OR “sun protection”] AND [“Latino” OR “Hispanic”] AND “beliefs.” Eligible papers were included in the final analysis after meeting the following inclusion criteria: (1) the records had to quantitatively examine and report sun protection beliefs in Hispanics, (2) the number of Hispanic participants in the sample had to be clearly specified, and (3) studies reporting differences in sun protection beliefs between Hispanics and other racial and ethnic groups were included in the review.Results. Of the 92 articles identified, 11 met inclusion criteria and addressed sun protection beliefs regarding skin cancer seriousness and susceptibility, and benefits and barriers of sun protection and skin cancer risk behaviors. Characteristics of studies and results were examined.Conclusion. There is insufficient evidence to determine a pattern of sun protection beliefs among Hispanics in the United States. More quality studies are needed which focus on sun protection beliefs in Hispanics.

scholarly journals Iraq from occupation to the risk of disintegration

2016 ◽  
Vol 9 (1) ◽  
pp. 27-48
Author(s):  
Sa'ad Naji Jawad
Keyword(s):  
United States ◽  
Final Analysis ◽  
The United States ◽  
American Occupation ◽  
Spheres Of Influence ◽  
The Us

This article discusses the longstanding and recent intentions of the United States to include Iraq into its spheres of influence. The American occupation of Iraq in 2003, however, not only changed the regime but also led to the destruction of the whole country. A most regrettable outcome of the US occupation of the country was the instigation of sectarian and ethnical differences, as well as the prolonged and continuing struggle between the centre and the peripheries. More harmful was the embodiment of all these negative and divisive phenomena into the permanent constitution of the country. In the final analysis, there have been many unsuccessful initiatives to solve the Iraqi dilemma throughout the last 13 years since the occupation. This article examines these initiatives in detail.

scholarly journals A Single-Arm, Open-Label, Expanded Access Study of Vemurafenib in Patients With Metastatic Melanoma in the United States

2014 ◽  
Vol 20 (1) ◽  
pp. 18-24 ◽  
Author(s):  
Lawrence Flaherty ◽  
Omid Hamid ◽  
Gerald Linette ◽  
Lynn Schuchter ◽  
Sigrun Hallmeyer ◽  
...  
Keyword(s):  
United States ◽  
Metastatic Melanoma ◽  
The United States ◽  
Open Label ◽  
Expanded Access

scholarly journals Hydroxyurea Utilization Patterns Among Sickle Cell Disease Patients in the United States, US FDA Sentinel Database

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2372-2372
Author(s):  
Rosanna Setse ◽  
Corinne Woods ◽  
Bindu Kanapuru ◽  
Talia Menzin ◽  
Justin Bohn ◽  
...  
Keyword(s):  
United States ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
The United States ◽  
Adult Patients ◽  
Cell Disease ◽  
The Us ◽  
Cumulative Duration ◽  
Study Population ◽  
Utilization Patterns

Abstract Background: Hydroxyurea, an antineoplastic agent, was approved in 1998 to reduce the frequency of sickle cell crises in adult patients with sickle cell disease (SCD). More recently in 2017, hydroxyurea was also approved for pediatric patients, 2 years of age and older with sickle cell anemia and recurrent moderate to severe painful crises. Prior to this, hydroxyurea had been used in the United States (US) in pediatric patients with SCD without a labeled indication. This study investigated hydroxyurea utilization patterns among pediatric and adult patients with SCD in the US prior to the pediatric approval using administrative claims data in the Sentinel database sponsored by the US Food and Drug Administration. Methods: Data from 17 health plans contributing to the Sentinel Distributed Database were queried from January 1, 2000 through September 30, 2015. Eligible patients had a claim with a diagnosis of SCD in any care setting at any time during the query period. SCD was identified using International Classification of Diseases, 9th Revision, Clinical Modification (ICD-9-CM) diagnosis codes. Patients were required to be enrolled in plans with both medical and drug coverage on the date of their SCD diagnosis. Patients who were dispensed hydroxyurea after diagnosis of SCD were identified as hydroxyurea users. Patients included in the sub-analysis evaluating hydroxyurea cumulative duration of therapy were required to be continuously enrolled with medical and drug coverage for at least 90 days prior to the initial hydroxyurea dispensing date, during which, gaps in coverage of up to 45 days were allowed. For this sub-analysis, follow-up began on the day of the first valid hydroxyurea dispensing and continued until the first occurrence of any of the following: 1) disenrollment; 2) death; 3) the end date of the data provided by each Data Partner; 4) the end of the last hydroxyurea exposure episode; or 5) the end of the query period. Cumulative duration of hydroxyurea therapy was calculated by summing days' supplies from outpatient pharmacy hydroxyurea dispensings for each patient. Results were stratified by age group (≤1, 2-5, 6-11, 12-17, 18-49, and 50+ years) and race. Results: A total of 95,606 patients with SCD were identified during the query period. Roughly half of patients (46.8%) were adults 18-49 years of age. Thirty-two percent (n= 30,900) were ≥50 years of age and 20.9% (n= 19,950) were ≤17 years of age. The study population included 40.3% African-American and 15.9% White patients. Race was unknown for 42.6% of the study population. Among pediatric SCD patients ≤1, 2-5, 6-11 and 12-17 years of age, hydroxyurea was dispensed to 4.1%, 7.7%, 16.5% and 16.0% of patients respectively. Among adults 18-49 years and ≥50 years, 18.4% and 4.7% respectively were dispensed hydroxyurea. The majority of pediatric hydroxyurea users were dispensed hydroxyurea for ≥ 325 days cumulatively. The median [inter quartile range] number of days of cumulative hydroxyurea exposure among patients ≤1, 2-5, 6-11, 12-17, 18-49 and 50+ years were 422 [139, 789], 420 [157, 836], 462 [156, 1145], 328 [120, 877], 204 [65, 505] and 253 [88, 599] days respectively. Conclusions: Although hydroxyurea was not approved for pediatric use in the US until 2017, for the period January 1, 2000 through September 30, 2015, the proportion of pediatric SCD patients 6-11 years and 12-17 years and adult SCD patients 18-49 years of age who were dispensed hydroxyurea were similar in the FDA Sentinel database. Among patients dispensed hydroxyurea, the durations of cumulative exposure were generally higher among pediatric compared to adult patients with SCD. The higher cumulative hydroxyurea exposure duration among pediatric SCD patients may reflect greater tolerability and adherence to hydroxyurea treatment in this population among other reasons. While this analysis evaluated utilization patterns prior to hydroxyurea approval for pediatric SCD patients, further research to evaluate any changes in hydroxyurea use patterns after the pediatric labeling would be useful. Disclosures No relevant conflicts of interest to declare.

scholarly journals Expanded Access and Right To Try Requests: The Community Oncologist's Experience

2021 ◽  
pp. OP.20.00569
Author(s):  
Marjorie E. Zettler ◽  
Yolaine Jeune-Smith ◽  
Bruce A. Feinberg ◽  
Eli G. Phillips ◽  
Ajeet Gajra
Keyword(s):  
United States ◽  
Clinical Trials ◽  
Treatment Options ◽  
The United States ◽  
Investigational Drug ◽  
Web Based ◽  
Expanded Access ◽  
Patients With Cancer ◽  
The Us ◽  
The Right

PURPOSE: For patients with cancer who have exhausted approved treatment options and for whom appropriate clinical trials are not available, access to investigational drugs through the US Food and Drug Administration's Expanded Access (EA) program has been an alternative since the program's inception more than 30 years ago. In 2018, federal Right To Try legislation was passed in the United States, creating a second pathway—one that bypasses the US Food and Drug Administration—to obtain unapproved drugs outside of clinical trials. The use of the two programs by community medical oncologists and hematologist-oncologists has not been studied. METHODS: Between October 2019 and February 2020, community oncologists-hematologists from across the United States completed web-based surveys about EA and Right To Try pathways for accessing unapproved drugs for their patients. Physicians were asked about their utilization of, and perceptions of, the two programs. RESULTS: Of the 238 physicians who completed the survey, 46% indicated that they had attempted to gain access to an investigational drug for a patient using the EA program, whereas 14% reported attempting to use Right To Try pathway to obtain an unapproved drug for a patient. Eighty-nine percent of those who tried to use the EA program reported success in obtaining the investigational drug versus 73% of those who attempted to use the Right To Try pathway. CONCLUSION: Our survey found that most community oncologists-hematologists were aware of both the EA and Right To Try pathways, but there is room for improvement in understanding and utilization of the programs.

The United States and the Iranian Nuclear Programme

2018 ◽  
Author(s):  
Steven Hurst
Keyword(s):  
United States ◽  
Decision Making ◽  
Domestic Politics ◽  
The United States ◽  
Nixon Administration ◽  
Comprehensive Plan ◽  
The Us ◽  
Nuclear Issue ◽  
The Relationship

The United States, Iran and the Bomb provides the first comprehensive analysis of the US-Iranian nuclear relationship from its origins through to the signing of the Joint Comprehensive Plan of Action (JCPOA) in 2015. Starting with the Nixon administration in the 1970s, it analyses the policies of successive US administrations toward the Iranian nuclear programme. Emphasizing the centrality of domestic politics to decision-making on both sides, it offers both an explanation of the evolution of the relationship and a critique of successive US administrations' efforts to halt the Iranian nuclear programme, with neither coercive measures nor inducements effectively applied. The book further argues that factional politics inside Iran played a crucial role in Iranian nuclear decision-making and that American policy tended to reinforce the position of Iranian hardliners and undermine that of those who were prepared to compromise on the nuclear issue. In the final chapter it demonstrates how President Obama's alterations to American strategy, accompanied by shifts in Iranian domestic politics, finally brought about the signing of the JCPOA in 2015.

‘To see oursels as ithers see us’: Reflections on Scottish Independence from the United States

2014 ◽  
Vol 23 (3) ◽  
pp. 381-388 ◽  
Author(s):  
Euan Hague ◽  
Alan Mackie
Keyword(s):  
United States ◽  
The United States ◽  
The Us ◽  
Scottish Independence ◽  
The Uk ◽  
Scottish Diaspora

The United States media have given rather little attention to the question of the Scottish referendum despite important economic, political and military links between the US and the UK/Scotland. For some in the US a ‘no’ vote would be greeted with relief given these ties: for others, a ‘yes’ vote would be acclaimed as an underdog escaping England's imperium, a narrative clearly echoing America's own founding story. This article explores commentary in the US press and media as well as reporting evidence from on-going interviews with the Scottish diaspora in the US. It concludes that there is as complex a picture of the 2014 referendum in the United States as there is in Scotland.

Palestine Unbound

2018 ◽  
Vol 47 (3) ◽  
pp. 130-134
Keyword(s):  
United States ◽  
Human Rights ◽  
Task Force ◽  
The United States ◽  
Donald Trump ◽  
Tel Aviv ◽  
The Us ◽  
Intention To Move ◽  
Arab Israeli Conflict ◽  
The U.S

This section, updated regularly on the blog Palestine Square, covers popular conversations related to the Palestinians and the Arab-Israeli conflict during the quarter 16 November 2017 to 15 February 2018: #JerusalemIstheCapitalofPalestine went viral after U.S. president Donald Trump recognized Jerusalem as the capital of Israel and announced his intention to move the U.S. embassy there from Tel Aviv. The arrest of Palestinian teenager Ahed Tamimi for slapping an Israeli soldier also prompted a viral campaign under the hashtag #FreeAhed. A smaller campaign protested the exclusion of Palestinian human rights from the agenda of the annual Creating Change conference organized by the US-based National LGBTQ Task Force in Washington. And, UNRWA publicized its emergency funding appeal, following the decision of the United States to slash funding to the organization, with the hashtag #DignityIsPriceless.

Sign in / Sign up

Export Citation Format

Share Document