An Update from the Benchmark Survey of phactMI™ Member Companies on Providing Medical Information in the Digital Space.

OBJECTIVE: The Pharma Collaboration for Transparent Medical Information (phactMI™) benchmarking survey of 32 pharmaceutical companies describes the use of technology by Medical Information Departments. METHODS: A survey was distributed to phactMI™ member companies in June 2022 and included 79 closed and open-ended questions. The survey's six sections included demographics, chatbot, social media, instant messaging applications, websites, and technology. RESULTS: Most Medical Information Departments have implemented innovative technology since 2019 with the main driver of remaining up-to-date. A total of 94% have a Medical Information website. Of those with a Medical Information website for healthcare professionals (HCPs), 97% allow for self-authentication. Most HCP-based websites have webforms for inquiries and 1-800 numbers, while only few offer video chat, chatbot, and the ability to identify local representatives. These websites also link to clinicaltrials.gov, publications, posters, and congress materials. Only 30% have a website for patients/caregivers. Most websites are discoverable by Google™. Awareness of Medical Information Websites occurs in a variety of ways, with most using multiple strategies to reach HCPs. There is wide variation in the technology platform used for the core functions of Medical Information. CONCLUSION: As technology continues to advance and omnichannel content remains a key objective, Medical Information needs to remain agile and transformative in their strategic and tactical planning and execution. Based on this benchmarking survey, the authors recommend that Medical Information Departments focus on expanding services for patients/caregivers, leverage digital innovations, expanding awareness, building efficiencies in workflow through technology, and continually improving website functionalities with innovative technologies.

Defining Insights.

BACKGROUND: Insights, when acted upon, can result in positive changes to the business, for HCPs, and ultimately for patients. Medical Information, as a customer facing function, is one of the groups that generate insights. Data and insights across different functions of an organization need to be compiled to provide a comprehensive view. The purpose of this paper is to develop a shared definition of insights and to provide a working guidance for the insight process. METHODS: Two surveys were conducted of the phactMI membership first to establish a shared definition of insights and then to benchmark current insight process. From this data and the shared experience of the working group a proposed guidance was developed. RESULTS: The developed definition of an insight is "An insight is the deeper understanding of the why behind trends of information that lead us to determine if an action is warranted". For the most robust outcomes, insight identification needs to be a cross functional activity. The proposed structured approach can be leveraged and customized for any organization and include the following five steps: INvestigate, Scrutinize, Identify, Take Action, and Enlighten (INSITE). CONCLUSION: The INSITE process provides a simple framework that should become routine for all Medical Information colleagues who are leading the work around insights. The process should be shared across all functions that participate in the insight generation process. This is another area where Medical Information can demonstrate leadership and highlight their value to the organization.

Medical Information Delivering Improved Customer Experience: A Guide.

Customer experience (CX) is essential in any business. In the pharmaceutical industry, the Medical Information Contact Center is a customer-facing unit that provides evidence-based, scientifically balanced information to healthcare professionals and patients in response to unsolicited inquiries. The purpose of this paper is to provide analysis and guidance for designing and measuring interactions in the Medical Information Contact Center to facilitate the delivery of a superior and continuously improving CX. Surveys were conducted to establish current trends in CX among a diverse group of CX professionals and members of phactMI, a non-profit collaboration of Medical Information leaders from the pharmaceutical industry. The top three observations from the CX professionals survey centered on establishing a clear CX strategy, use of technology, and frequency of sharing results. Three potential areas for improvement focus on CX strategy, measurements of CX, and sharing of results. An analysis of quality monitoring results of customer interactions in the pharmaceutical industry from Centerfirst, a contact center quality monitoring service provider, was also reviewed. This analysis found a positive correlation between CX and three agent skills: taking the lead, empathy, and strong compliance skills. Based on these results, a CX guide was developed and specifically tailored for the pharmaceutical industry. This tool may be used to help identify, assess, and possibly improve CX.

The Medical Information Scientific Process: Define, Research, Evaluate, Synthesize, and Share (DRESS).

Medical information (MI) professionals are primarily responsible for researching and responding to unsolicited requests for information on their company's product(s). In an effort to set a standard for quality, the Pharma Collaboration for Transparent Medical Information (phactMI) created a code of practice for the provision of medical information to healthcare professionals. This code introduced the term "MI science skills" to describe the expertise required to perform the duties of an MI professional. These skills can be summarized by the acronym DRESS. In order to effectively and efficiently respond to an unsolicited request for information, the MI professional essentially follows five steps: define the question, research the topic, evaluate the evidence, synthesize a response, and share the answer. As this approach mirrors the scientific process for data generation, MI scientist may be a more apt description for this role. This paper explains the rationale behind the term MI scientist and the skills associated with each component of the DRESS approach.

Enhancing Patient Care II: The Clinical Impact of Medical Information Services.

BACKGROUND: Understanding the ways that healthcare providers (HCPs) utilize medical information received from the pharmaceutical industry is important so that the information can be tailored and customized to meet the HCPs needs. Additionally, this understanding supports the value of the information provided. The purpose of this study was to collect opinions of HCPs who recently requested information from a manufacturer's Medical Information (Med Info) Service. HCPs provided their opinions on the perceived quality, relevance, impact on patient care, and intended usage of information. METHODS: HCPs who recently requested medical information from one of eight participating companies received a Survey Monkey link in the information response. Data collected included demographics, perceived quality, relevance, impact on patient care, and intended usage of the information. Data were analyzed via descriptive statistics. RESULTS: Over a 14-month period, 246 HCPs responded to the survey. Eight companies participated in the survey. Customer responses to the survey ranged from 2 to 97 per company. A total of 99 pharmacists, 68 physicians, 22 registered nurses, 21 nurse practitioners, 8 physician assistants, and 28 others participated in the survey. Most HCPs (208/227, 92%) contacted the company Med Info Group 1-5 times in the last six months and 67% (159/238) had been in practice greater than 10 years. Most HCPs rated the following quality areas as a 4 or 5 on a 5-point Likert scale: timeliness (195/225, 87%), trustworthiness (189/221, 86%), conciseness (185/221, 84%), clarity (180/222, 81%), relevance (178/223, 80%), and completeness (173/222, 78%). The most common reason for contacting Med Info Services was to advance knowledge or education (110/228, 48%). Additional reasons were at the point of care (60/228, 26%), for a specific patient (not at point of care) (60/228, 26%), and to reflect on a treatment decision (59/228, 26%). The relevance of the information provided was utilized for the HCPs own education (99/226, 44%), shared with peers (91/226, 40%), or used for future treatment decisions (88/226, 39%). The information provided enhanced patient care by enabling the HCPs to educate patients more effectively (86/222, 39%), efficacy of treatment regimen was enhanced (70/222, 32%), or other positive impact (65/222, 29%). CONCLUSION: The opinions of HCPs who are using Medical Information Services are overall positive. All the quality indicators were rated as a 4 or 5 by the majority of HCPs, with the lowest in completeness (173/222, 78%) and the highest in timeliness (195/225, 87%). Medical Information Services were utilized to advance knowledge/education of the HCP, followed closely by the care of a current or future patient. However, when queried on the relevant use of the information in their practice, the most common answers were for their own education or to share with peers. The impact on patient care was focused on enabling the HCP to educate patients more effectively. The value of medical information is difficult to quantify. Understanding the quality assessment, utilization, and the impact on patient care by HCPs can provide a broad descriptor of value. This study supports the value of the medical information responses provided by pharmaceutical companies to HCPs in their practice(s).

Emerging Therapies for Peanut Allergy.

Peanut allergy (PA) is a food allergy that causes an IgE-mediated type I hypersensitivity reaction. PA has become an increasing public health burden, with 2% of American children reported to have this condition in 2010. Current guidelines recommend allergen avoidance, patient education, and administration of H1 antihistamines, ß2-agonists, or epinephrine based on the severity of reaction. In this review article, emerging therapies for PA are evaluated for their potential role in treating PA. Oral, epicutaneous, and sublingual immunotherapies have completed clinical trials with promising efficacy. In particular, Palforzia (AR101) is an oral immunotherapy that received Food and Drug Administration (FDA)-approval in January 2020 and Viaskin Peanut is an epicutaneous immunotherapy with an anticipated FDA decision date by August 5, 2020. Furthermore, adjuvant combinations with either probiotics or anti-IgE receptor antagonists have shown an improved efficacy and safety profile compared to oral immunotherapy alone. However, immunotherapy-induced adverse reaction rates are high due to the risks associated with intentional allergen exposure. These results suggest that peanut immunotherapy has a promising role in the treatment of PA, although further studies are needed before its incorporation into standard of care.

Preferences for Accessing Medical Information in the Digital Age: Health Care Professional Survey.

BACKGROUND: Health care professionals (HCPs) routinely have questions concerning the medications they are recommending. There are numerous resources available; however, each has its own advantages and disadvantages. OBJECTIVE: The purpose of this survey was to gain knowledge of the preferred methods and sources HCPs use to obtain information concerning medications. METHODS: A total of 511 HCPs (202 physicians, 105 pharmacists, 100 advance practice nurses, 53 registered nurses, and 51 physician assistants) were surveyed through a third-party market research firm. All participants were practicing in the United States. Individuals working for a pharmaceutical company were excluded. The survey collected demographics, frequency of searching medical information, types of questions searched, sources of medical information, and rationale for preferred and nonpreferred sources of medical information. Use of medical information resources were rated on a 5-point ordinal scale. Data were analyzed with descriptive statistics. RESULTS: Of the 511 respondents, 88.5% (452/511) searched for medical information either daily or several times per week. The most common questions involved dosing and administration, drug-drug interactions, adverse events and safety, clinical practice guidelines, and disease state information. The main rationale for using specific medical websites or apps and general online search engines frequently or very frequently was ease of use (medical websites or apps: 269/356, 75.6%; general online search engines: 248/284, 87.3%). Accuracy was the main rationale for frequent or very frequent use of medical literature search databases (163/245, 66.5%), prescribing labels or information (122/213, 57.3%), and professional literature (120/195, 61.5%). The main reason for rarely or never using specific medical websites or apps and medical literature search databases was unfamiliarity (medical websites or apps: 16/48, 33%; medical literature search databases: 35/78, 45%); for general online search engines, inaccuracy (34/54, 63%); and for prescribing labels or information and professional literature, excessive time (prescribing labels or information : 54/102, 52.9%; professional literature: 66/106, 62.3%). The pharmaceutical company was sometimes used as a resource for medical information. When the medical information department was used, the call center and the website were considered thorough and complete (call center: 14/25, 56%; website: 33/55, 60%). However, the rationale for not using the call center was the time required (199/346, 57.5%) and the website being unfamiliar (129/267, 48.3%). CONCLUSIONS: The driving forces in the selection of resources are accuracy and ease of use. There is an opportunity to increase awareness of all the appropriate resources for HCPs which may aid in their daily clinical decisions. Specifically, pharmaceutical company medical information departments can help fulfill this need by addressing two major challenges with use of the pharmaceutical company: lack of awareness of medical information services and the speed at which responses are disseminated. Overall, there is lack of understanding or appreciation of the range of pathways to obtain published information and knowledge from pharmaceutical company medical information services. Among the many challenges resource champions will face are the ability to effectively make resources and their platforms accessible, known, and useful to the scientific community.

Proposed Best Practice Guidelines for Scientific Response Documents: A Consensus Statement from phactMI.

The Medical Information Department of a pharmaceutical manufacturer provides written scientific responses to unsolicited requests from healthcare providers for information on products that extends beyond the product labeling (off-label). These scientific response documents are non-promotional, evidence-based, and scientifically balanced, conforming with internal pharmaceutical manufacturer's procedures and the Food and Drug Administration (FDA) Draft Guidance on Responding to Unsolicited Requests for Off-Label Information. Members of phactMI™ developed this proposal to offer best practices for content generation of scientific response documents. Scientific response documents review available literature to respond to an unsolicited request; therefore, they are similar in nature to systematic reviews. The sections and elements identified in this proposed best practice guidelines for scientific response documents are based on an adaptation of the sections and elements of systematic reviews. The sections of a scientific response document should include a restatement of the unsolicited request (title); a structured summary (abstract); approved indications, black box warnings, and background information when appropriate (introduction); the literature search information and study selection (methods); summation of data from clinical trials, meta-analysis, case reports, and/or real world evidence, as appropriate (results); treatment guidelines, if applicable and available (discussion); and references. Elements for each section should be included in a scientific response document as appropriate, as some elements are not necessary in some documents, based on the question. These elements were selected for inclusion to address any potential concerns of bias and transparency and reflect the intent that scientific response documents should be non-promotional, accurate, truthful, free of commercial bias, scientifically balanced, and evidence based.

Presentation of scientific poster information: Lessons learned from evaluating the impact of content arrangement and use of infographics.

BACKGROUND: Scientific poster content and delivery methodology have evolved in recent years. The objective of this observational cross-sectional survey-based study was to evaluate pharmacists' preferences of two different scientific poster formats conducted at a conference in May 2017. Two posters on the same topic were developed and presented utilizing different formatting; one in the traditional, text-based format and the other in an infographic-based format. Study participants (n = 61) included 23 (38%) preceptors and 37 (61%) residents. Preceptors and residents scored clarity and comprehensibility similarly for both poster formats respectively, but rated aesthetic appeal higher for the infographic format. Both groups found more detailed information to be missing from the infographic poster format. Overall, residents did not prefer one poster format to another, while preceptors overwhelmingly preferred the infographic poster format over the traditional poster format. IMPACT: Several key confounders limit the interpretations of the study results. These confounders include lack of large and well-distributed sample size, inability to control for the effect of preference on comprehension scores, and differences between resident and preceptor experience that may ultimately influence preferences and results. RECOMMENDATIONS: In repeating this study, investigators should consider capturing a national and larger sample size to increase applicability of results, design questions to assess comprehension, and collect participant baseline characteristics. DISCUSSION: Variations in preferences and perceptions for optimal scientific poster content and design among pharmacists exist. The findings of this study suggest infographic poster formats are more aesthetically appealing, but demonstrate similar clarity and comprehensibility as a traditional poster format.

Comparison of Postdoctoral Pharmacy Training Programs: Drug Information Residencies and Medical Information Fellowships.

Pharmacy graduates interested in postdoctoral training opportunities in drug information or Medical Information have the option to pursue either a fellowship within the pharmaceutical industry or a clinical residency. Limited resources exist for pharmacy students to gain insight into the differences between drug information (DI) and medical information (MI) training programs. The purpose of this project is to identify available opportunities for postdoctoral training in DI or MI disciplines and to identify similarities and differences between them. DI residencies and MI fellowships were identified by examination of the American Society of Health-System Pharmacists (ASHP) and the American College of Clinical Pharmacy (ACCP) residency directories, the ASHP Personal Placement Service (PPS) index, and online keyword searching for nonindexed programs. The authors investigated individual programs via publicly available information to classify core responsibilities and skill sets developed; a total of 24 DI residency programs and 33 MI fellowships focusing or containing at least 1 component in medical information were evaluated. All ASHP-accredited DI residencies offered teaching, formulary management, adverse drug reaction and medication error reporting, drug utilization evaluation, and policy development as professional growth opportunities. The most commonly encountered development activities in MI fellowships were creating standard response documents, collaborating with cross functional teams, teaching or precepting, and reviewing promotional materials. Institutions and health care providers (HCPs) are the primary recipients of DI services whereas MI services respond to consumers, payors, HCPs, and external organizations. Employment prospects commonly overlap between the 2 training programs.

Publish or perish: Success with publication in pharmacy residency training.

BACKGROUND AND PURPOSE: Pharmacy residency training standards require development of medical writing skills. These skills are fundamental to pharmacy clinicians and scholars alike. Despite this requirement, new practitioners and seasoned clinicians frequently struggle with scholarly development and manuscript generation for a variety of reasons, ranging from lack of experience or familiarity with the process of peer-review submission to time constraints. EDUCATIONAL ACTIVITY AND SETTING: We describe a process for fostering pharmacy resident scholarship and publication utilized at Robert Wood Johnson University Hospital, including the stages of the project development process and corresponding responsibilities during each phase of the manuscript submission process. From identification of interest, to project and manuscript development, review, submission, revision, and reflection, our postgraduate pharmacy residents receive guidance and structure from a preceptor mentor to usher them through this experience for the first time. FINDINGS: The program has had success utilizing this structured approach to supporting residency publication efforts, with the preparation of 23 manuscripts that have resulted in peer-reviewed publications from 28 residents graduating between 2013 and 2017. This results in a resident authorship rate of 82%. SUMMARY: Although medical writing and manuscript development may not be an intuitive process, it is imperative that preceptors and individuals interacting with postgraduate pharmacy trainees consider exposing their trainees to this process, with foresight into thinking about a structured approach to publication at the onset of project development.

Use of nitroglycerin ointment to treat primary and secondary Raynaud's phenomenon: a systematic literature review.

Raynaud's phenomenon (RP) is a microvascular condition in which reversible ischemic attacks occur in the extremities. Due to the unpredictable nature of these attacks, pharmacologic agents that can be administered on as-needed basis are currently being sought after. Topical nitrates are well suited for as-needed use, and several different formulations have been studied for the treatment of RP, including ointments, gels, patches, and tapes. However, these different dosage forms are not all equal in terms of safety and efficacy, and not every one is commercially available for use in clinical practice. Nitroglycerin ointment is commercially available, and it has less systemic side effects than other topical formulations. Since its role in the treatment of RP has not yet been completely established, we performed a systematic search of Medline, Embase, and the Cochrane Central Register of Controlled Trials to evaluate its safety and efficacy. A total of 1125 studies were identified, and 7 were included in our review. Although the included studies used different measures of efficacy, the majority reported positive responses to nitroglycerin ointment. The benefit of nitroglycerin ointment in the treatment of RP may be further realized through more robust investigation.

Transdermal estradiol for the management of refractory uremic bleeding.

PURPOSE: The efficacy and thrombogenicity of transdermal estradiol in the management of refractory uremic bleeding in adults are examined. SUMMARY: Platelet dysfunction from chronic kidney disease may induce uremic bleeding. This type of bleeding may involve the skin, oral and nasal mucosa, gingivae, respiratory system, and gastrointestinal or urinary tract. While the mainstay of treatment for uremic bleeding primarily involves dialysis and use of prohemostatic agents such as desmopressin and erythropoiesis-stimulating agents, certain patients may experience bleeding refractory to these interventions. In this clinical scenario, a weak conditional recommendation (grade 2C) supporting transdermal estradiol as a therapy of last resort exists. Limited data suggest that transdermal estradiol may reduce bleeding time and transfusion requirements in dialysis patients with recurrent episodes of hematochezia, gastrointestinal telangiectasia, and hematomas. The management of uremic bleeding will require long-term therapy, and case reports have documented the safe use of transdermal estradiol for up to 25 months. Oral conjugated estrogens increase the risk of deep vein thrombosis in women; however, the transdermal route of administration has been associated with a lower incidence of venous thromboembolism and stroke relative to oral estrogen and, in some studies, its associated risk of thrombosis is not significantly different when compared with placebo. CONCLUSION: Patients who are refractory to routine interventions for uremic bleeding may benefit from transdermal estrogen despite the limited data. Extended therapy with low-dose transdermal estrogen (≤50 µg daily) may provide a hemostatic benefit that outweighs thrombotic risk.

Disaster Preparedness: Biological Threats and Treatment Options.

Biological disasters can be natural, accidental, or intentional. Biological threats have made a lasting impact on civilization. This review focuses on agents of clinical significance, bioterrorism, and national security, specifically Category A agents (anthrax, botulism, plague, tularemia, and smallpox), as well as briefly discusses other naturally emerging infections of public health significance, Ebola virus (also a Category A agent) and Zika virus. The role of pharmacists in disaster preparedness and disaster response is multifaceted and important. Their expertise includes clinical knowledge, which can aid in drug information consultation, patient-specific treatment decision making, and development of local treatment plans. To fulfill this role, pharmacists must have a comprehensive understanding of medical countermeasures for these significant biological threats across all health care settings. New and reemerging infectious disease threats will continue to challenge the world. Pharmacists will be at the forefront of preparedness and response, sharing knowledge and clinical expertise with responders, official decision makers, and the general public.

Differentiating predatory scholarship: best practices in scholarly publication.

OBJECTIVE: The intent of this article is to define predatory publishing, identify the risks and costs associated with publishing scholarship with these types of organizations and to provide recommendations for best practices how a potential author can protect themselves against predatory organizations. METHODS: A thorough review of the literature concerning predatory publishing was conducted and gleaned for best practices along with the authors' experiences. KEY FINDINGS: Pharmacy scholars and researchers worldwide recognize the virtues of the open access (OA) publication system, which is intended to freely disseminate research electronically, stimulate innovation and improve access to scholarship. Both subscription-based and OA publication systems, however, have potential areas of conflicts, including coordination of the peer-review process and the potential for the publisher to capitalize on selling the commodity in a capitalistic society. The intent of OA is welcomed; however, publishers are still in a business and profits need to be made. It is by the exploitation of the model that has given rise to a small but growing subset known as predatory publishers. CONCLUSIONS: Pharmacy researchers and clinicians alike need to be aware of predatory organizations, both publishers and meeting organizers, when seeking a venue to publish their own scholarly research. Additionally, this knowledge is critical when evaluating medical literature in providing direct patient care services to assure the best available evidence is utilized.

A Review of the Safety and Efficacy of Vaccines as Prophylaxis for Clostridium difficile Infections.

This review aims to evaluate the literature on the safety and efficacy of novel toxoid vaccines for the prophylaxis of Clostridium difficile infections (CDI) in healthy adults. Literature searches for clinical trials were performed through MEDLINE, ClinicalTrials.gov, and Web of Science using the keywords bacterial vaccines, Clostridium difficile, and vaccine. English-language clinical trials evaluating the efficacy and/or safety of Clostridium difficile toxoid vaccines that were completed and had results posted on ClinicalTrials.gov or in a published journal article were included. Six clinical trials were included. The vaccines were associated with mild self-reported adverse reactions, most commonly injection site reactions and flu-like symptoms, and minimal serious adverse events. Five clinical trials found marked increases in antibody production in vaccinated participants following each dose of the vaccine. Clinical trials evaluating C. difficile toxoid vaccines have shown them to be well tolerated and relatively safe. Surrogate markers of efficacy (seroconversion and geometric mean antibody levels) have shown significant immune responses to a vaccination series in healthy adults, indicating that they have the potential to be used as prophylaxis for CDI. However, more research is needed to determine the clinical benefits of the vaccines.

Evidence for the Use of Epoprostenol to Treat Raynaud's Phenomenon With or Without Digital Ulcers.

OBJECTIVE: To review the evidence for using intravenous (IV) epoprostenol to treat Raynaud's phenomenon (RP). DATA SOURCES: The databases MEDLINE (1946 to March 2016), PubMed, and International Pharmaceutical Abstracts were searched using the terms epoprostenol, Flolan, Raynaud's disease, and CREST syndrome. Further literature sources were identified by reviewing article citations. STUDY SELECTION AND DATA EXTRACTION: All English-language, clinical trials and case series evaluating IV epoprostenol for the management or treatment of RP were included. Lower-quality evidence were incorporated due to limited information. DATA SYNTHESIS: Seven small uncontrolled studies/case series, 1 small placebo controlled study, and 1 larger randomized trial were identified and included. There was no consistent measurement of efficacy utilized, but improvements in hand temperature, RP attack duration and frequency were commonly associated with IV epoprostenol treatment (5 trials). There were conflicting data regarding effect sustainability, with 5 trials showing long-term effects and 3 showing immediate effects. Fewer ischemic ulcers developed during treatment with IV epoprostenol in 1 trial compared to conventional treatment. Ulcer healing ocurred in 2 trials. Common adverse effects included hypotension, headache, flushing, gastrointestinal symptoms, and jaw pain. CONCLUSIONS: Available evidence supports the use of IV epoprostenol for treatment of severe RP in patients refractory or intolerant to standard therapies. The dose, titration schedule, and duration of IV epoprostenol utilized in studies varied, but a conservative approach to initiation should be considered. Patients who do not respond to intermittent infusions and have severe digital ischemia may require more aggressive regimens.

The role of colesevelam hydrochloride in hypercholesterolemia and type 2 diabetes mellitus.

OBJECTIVE: To evaluate the safety and efficacy of colesevelam hydrochloride for the treatment of hypercholesterolemia and type 2 diabetes mellitus. DATA SOURCES: Literature retrieval was accessed through MEDLINE/PubMed (1950-March 2010), Web of Science (1980-March 2010), and International Pharmaceutical Abstracts (1977-March 2010) using the terms colesevelam, dyslipidemia, hypercholesterolemia, and type 2 diabetes mellitus. References from publications identified were reviewed for additional resources. In addition, abstracts presented at the most recent (2009) American Diabetes Association, American Association of Clinical Endocrinologists, and European Association for the Study of Diabetes annual meetings were searched for relevant original research. STUDY SELECTION AND DATA EXTRACTION: All articles in English identified from the data sources were evaluated. All relevant studies evaluating the safety and efficacy of colesevelam in hypercholesterolemia and/or type 2 diabetes mellitus were included. Priority was placed on data obtained from human randomized controlled trials. DATA SYNTHESIS: Seventeen clinical trials were reviewed and evaluated. Of the clinical trials evaluating colesevelam in hypercholesterolemia, 3 evaluated monotherapy, 4 evaluated combination therapy with hydroxymethylglutaryl coenzyme A (HMG-CoA) reductase inhibitors, and 6 evaluated combination therapy with other lipid-lowering therapies. In the type 2 diabetes mellitus trials, colesevelam was evaluated in combination with metformin, sulfonylureas, insulin, and rosiglitazone and sitagliptin. A review of the clinical trials provided evidence that colesevelam monotherapy effectively reduces low-density lipoprotein cholesterol (LDL-C). Additionally, the use of colesevelam in combination with other lipid-lowering therapies further reduces LDL-C. Colesevelam also effectively reduces hemoglobin A(1c) in patients with type 2 diabetes mellitus. The safety and tolerability of colesevelam appear to be improved from that of older-generation bile acid sequestrants, with adverse effects similar to those with placebo in monotherapy and type 2 diabetes mellitus trials. CONCLUSIONS: Colesevelam is a safe and effective option for the treatment of hypercholesterolemia and type 2 diabetes mellitus. It can fulfill a useful role in combination with HMG-CoA reductase inhibitors for hypercholesterolemia and should be considered in patients with type 2 diabetes mellitus with concomitant hypercholesterolemia.

Enhancing survival outcomes in the management of patients with higher-risk myelodysplastic syndromes.

The myelodysplastic syndromes (MDS) are a collection of clonal myeloid neoplasms characterized by bone marrow failure and cytopenias. Patients classified with higher-risk disease include those with intermediate-2 or high-risk disease as classified by the International Prognostic Scoring System (IPSS). These patients represent 29% of the MDS population and were originally reported to have a median survival without therapy of only 0.4 for high-risk patients and 1.1 years for intermediate-2 risk patients. The most significant treatment goals in these patients involve prolonging the time to acute myeloid leukemia progression and extending overall survival. Quality of life, symptom control, and transfusion independence are also important. The National Comprehensive Cancer Network guidelines divide treatment options for this population into high- and low-intensity therapies. High-intensity therapies include cytarabine-based remission induction chemotherapy and hematopoietic stem cell transplantation. Transplantation is the only treatment option with the ability to cure; however, many MDS patients may not qualify due to age or comorbidities. Low-intensity therapies include the DNA methyltransferase inhibitors azacitidine and decitabine. Recently, azacitidine demonstrated the ability to extend survival by as much as 74% despite a modest complete response rate. This review examines the classification and diagnosis of higher-risk MDS patients, the management goals for these patients, clinical experience involved with treatments, guidelines, and recommendations for therapeutic options.