Free CME

In this online CME self-learning program:

Gaucher disease is characterized by a deficiency of the lysosomal enzyme glucocerebrosidase, resulting in the accumulation of sphingolipids throughout the body but most manifesting prominently in the bones. One of its first complications is the chronic anemia and a persistent bleeding risk. Another is the hepatosplenomegaly, which may be a part of the initial clinical presentation, as may the anatomical abnormalities of bone deformities and stunted growth.

Fabry disease is characterized by a deficiency of the glycoside hydrolase enzyme alpha galactosidase A, resulting in the accumulation of the glycolipid globotriaosylceramide throughout the body, particularly prominently in the blood vessels, which ultimately leading to multi-organ dysfunction and the patient’s premature death. Early symptoms, which occur during childhood, involve pain and may include Raynaud phenomenon, paresthesias, and arthralgia in the extremities and proximal limbs, as well as impaired gastrointestinal emptying, resulting in abdominal pain, diarrhea, early satiety, postprandial bloating, nausea, and vomiting. In adulthood, the disease’s impact spreads beyond and begins to affect the cardiac and renal systems^.

Annual meetings of large, national, professional societies offer an opportunity for HCPs to get a first glimpse at study results that have the potential to impact practice as provide a forum for an exchange of ideas and practices between thought leaders and less distinguished practitioners. Nevertheless, many HCPs will be unable to attend these conferences, justifying the creation of CME that summarize the major findings presented at these major meetings.

Target Audience:

HCPs including: MD specialists, pediatricians, primary care physicians; geneticists; physician assistants, nurse practitioners, nurses, and pharmacists; and any other HCPs with an interest in or who may clinically encounter patients with Lysosomal Storage Disorders.

By the end of the session the participant will be able to:

• Recall the clinical manifestations of representative lysosomal storage disorders (LSDs).
• Describe how enzyme replacement therapy (ERT) is being improved to address unmet therapeutic needs.
• List emerging therapies for patients with LSDs who have previously been treated with ERTs and apply them to patient cases.
• Discuss the findings of trials of gene replacement therapy particularly for LSDs that affect the central nervous system (CNS).

Target Audience:

HCPs specializing in: Infectious diseases, HIV, and internal medicine; physician assistants, nurse practitioners, and pharmacists who practice in infectious disease; and any other healthcare professionals with an interest in or who clinically encounter patients with HIV.

By the end of the session the participant will be able to:

• Discuss highlights and changes within the most current HIV treatment guidelines, including the roles of fixed-dose combinations, single-tablet regimens, and once-daily treatment options.
• Evaluate a treatment plan and suggest modifications for improvement, taking the following into account: patient preference, adherence, pill burden, comorbidity level, and drug-drug interactions.
• Develop a treatment plan that optimizes safety and efficacy using patient cases.
• Describe the challenges and barriers to care associated with treating patients with HIV.

Each year, an estimated 170,000 cases of prostate cancer are diagnosed. Although early prostate cancer may be cured with surgery or radiation therapy, more than 50% of men will experience recurrence after definitive treatment. The use of androgen deprivation therapy (ADT) for intermediate-risk, high-risk, and recurrent prostate cancer has greatly increased in the last 15 years, extending 10-year survival by nearly 25% compared with observation alone. New treatment options for advanced prostate cancer have further improved survival and increased the number of patients living with non-metastatic and metastatic castration-resistant prostate cancer (nmCRPC and mCRPC). Despite the established improvements in survival associated with hormonal treatments, ADT has been associated with well-characterized negative effects on bone health. Indeed, bone metastases and subsequent skeletal-related events (SREs) are the primary driver of both morbidity and mortality among people with CRPC. In a study of 2234 men aged 65 years or older with a diagnosis of CRPC, 40% experienced SREs over a median follow-up of 10.6 months. Maintaining bone health in patients with CRPC requires routine monitoring and proactive management.

Target Audience:

The following healthcare professionals: urologists, medical and radiation oncologists, primary care physicians, nuclear medicine specialists and radiologists, and endocrinologists; physician assistants, nurse practitioners, and pharmacists who treat prostate cancer; and any other healthcare professionals who commonly care for patients with prostate cancer.

By the end of the session the participant will be able to:

• Recall the relative efficacy and safety of presently available agents used to for bone health and fracture prevention in patients with prostate cancer
• Describe the identification and management of disease and intervention adverse effects on patients
• Using details in a patient case, recommend an appropriate pharmacotherapeutic treatment plan for the purpose of preventing SREs in patients with prostate cancer

Activity Description / Statement of Need:

In this online, self-learning activity:

Glaucoma, a heterogeneous disease leading to progressive damage to the optic nerve, causes irreversible vision loss and affects approximately 64 million people worldwide.The prevalence of glaucoma is increasing, and more than 100 million people will have glaucoma by 2040. Approximately half of all individuals with glaucoma are unaware of their condition due to the asymptomatic nature of the disease. Although the incidence of glaucoma-related blindness has decreased over the last 20 years, thanks to effective management strategies, 13% to 40% of people with glaucoma still develop unilateral or bilateral blindness, typically at a rate of 1.1% per year.

Target Audience:

The following healthcare professionals: ophthalmologists; physician assistants, nurse practitioners,  and any other healthcare professionals with an interest in or who clinically encounter patients with glaucoma.

Alpha 1-antitrypsin deficiency (AATD), characterized by low serum levels of the serine protease inhibitor alpha-1 antitrypsin (AAT), is a genetic disorder resulting in destruction of lung structures. Reduced levels of AAT result in overactivity of neutrophil elastase, which destroys connective tissue within the lung and causes degradation of alveoli, reduced pulmonary elastic recoil, and airflow. Breakdown of the alveoli eventually manifest as emphysema or other forms of chronic lung disease, including chronic obstructive lung disease (COPD). Other complications associated with AATD include liver disease, panniculitis, and vasculitis. The most common cause of death in patients with severe AATD is respiratory failure, which accounts for 45 to 72% of deaths. Smoking, occupational hazards such as firefighting, and high levels of cumulative exposure to pollution accelerate the rate of lung function decline in people with AATD. AATD is estimated to affect one out of every 2,000 to 5,000 individuals, mainly of North European or Iberian ancestry, with a global prevalence of over 3.4 million affected individuals.

Target Audience:

The following healthcare professionals: pulmonologists and primary care physicians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in pulmonology and internal medicine; and any other healthcare professionals with an interest in or who clinically encounter patients with AATD.

By the end of the session the participant will be able to:

• Summarize the present state of awareness of AATD among healthcare professionals and its attendant impact on patient care.
• Describe the best time to test a patient for AATD and whether to conduct a family screening.
• Apply best treatment practices to patients with AATD in various clinical scenarios.
• Describe the challenges associated with the optimal diagnosis and treatment of AATD.

Inflammatory bowel disease (IBD) is a broad term that includes many different forms of inflammatory bowel conditions, the most common of which are ulcerative colitis (UC) and Crohn’s disease (CD), the former of which exclusively affects the colon and rectum. UC occurs more frequently than CD, with an incidence of 1.2 to 20.3 cases per 100,000 persons per year and a prevalence of 7.6 to 246.0 cases per 100,000 per year, as compared to 0.03 to 15.6 and 3.6 to 214.0 per 100,000 for CD. Risk factors include characteristics common in westernized environments and lifestyles, including smoking, diets high in fat and sugar, medication use, stress, and high socioeconomic status.

Target Audience:

HCPs specializing in: gastroenterology, internal medicine; nurse practitioners, physician assistants, and pharmacists who specialize in gastroenterology; and those who otherwise commonly care for or clinically encounter patients with UC.

By the end of the session the participant will be able to:

• Describe the burden of disease UC represents to patients.
• Identify the present treatment options currently available for management of UC and apply them to patient cases using evidence-based medicine.
• Identify new and emerging therapies for the treatment of UC.
• Evaluate a treatment plan for a specific patient based on severity of UC to optimize safety, efficacy, and cost-efficacy, suggesting modifications for improvement.

Cystic Fibrosis (CF) is a genetic disease that affects nearly 70,000 people worldwide with more than 90% of patients diagnosed of Caucasian descent and a median lifetime survival remains a mere 43.6 years. CF is caused by an autosomal recessive mutation in the CF transmembrane regulator (CFTR) gene, which controls the other chloride and sodium channels at the cell surface and is found in the lungs, liver, pancreas, intestine, sweat duct, and epididymis. The primary organs in which the disease manifests clinically are the pancreas, leading to malabsorption of nutrients, and the lungs due to the accumulation of thick, sticky mucous that contributes to airway obstruction. CF causes several clinical complications, including recurrent pulmonary infections, nasal polyps, CF-related diabetes, fat-soluble vitamin deficiencies, acid reflux, and liver failure.

Target Audience:

The following HCPs: pulmonologists, pediatricians, gastroenterologists and primary care physicians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in the aforementioned areas of specialty; and any other healthcare professionals with an interest in or who clinically encounter patients with CF.

By the end of the session the participant will be able to:

• Describe the pathophysiology of CF such that it might inform treatment mechanisms.
• Identify the currently available and emerging pharmacotherapeutic treatments for the management of CF and apply them to patient cases using evidence-based medicine.
• Describe newly approved and investigational therapies in development for CF.
• Evaluate an ongoing treatment plan for a specific patient with CF to optimize safety and efficacy, suggesting modifications for improvement, including the management of complications.

Alzheimer Disease (AD) is a degenerative disease that most commonly affects the elderly, although it is occasionally detected as early as middle age. AD accounts for over half of all diagnosed dementia, the prevalence of which is increasing. Once there is a diagnosis of probable AD, one must determine which pharmacotherapy, if any, is most appropriate for treatment of the patient. The literature suggests that gaps in care on the part of healthcare professionals exist. While some physicians continue to use the antiquated, nonspecific term “senile dementia” as a descriptive diagnosis of AD, perhaps suggesting a lack of understanding of the gravity of the disease state or its associated pathophysiology, other report feeling uncertain at times about now to best diagnosis of the disease, particularly in its very early stages. Moreover, a number of different practice guidelines have been updated recently, and HCPs are often unable to keep up with the publishing of literature and evolution of clinical practice.

Target Audience:

Healthcare professionals specializing in: neurology, gerontology, internal medicine, palliative care, or those who otherwise commonly care for patients with AD or who frequently encounter them or their caregivers in practice.

By the end of the session the participant will be able to:

• Describe what is presently known about the pathophysiology of AD.
• Describe the challenges associated with diagnosis and treatment of ADDiscuss the neuropathophysiology of AD and how it relates to presently available AD treatments mechanisms.
• Identify the present treatment options currently available for management of AD and apply them to patient cases using evidence-based medicine.
• Describe emerging drug therapies in the treatment of AD.
• Evaluate a treatment plan for a specific patient based on degree of AD to optimize safety and efficacy, suggesting modifications for improvement.

Osteoporosis is a disease common among elderly patients and is increasing in frequency as senior citizens begin to represent a larger share of the US population. In the US, osteoporosis is associated with about 2 million broken bones each year, leading to over 500,000 hospitalizations, 800,000 emergency room visits, and 200,000 nursing home placements. By 2040, osteoporosis is expected to cause 3.2 million fractures per year in the United States at a cost of over $95 billion. Despite the morbidity and mortality associated with osteoporosis, practice gaps related to suboptimal screening, risk assessment, and management practices have led to underdiagnosis and undertreatment of this condition. Osteoporosis screening may identify people at increased risk of low-trauma fracture who may benefit from interventions to minimize risk. The US Preventive Services Task Force (USPSTF) recommends screening for osteoporosis with bone mineral density (BMD) testing in all women 65 years or older and in postmenopausal women younger than 65 years but at increased risk of osteoporosis. Risk for osteoporosis should be determined by a formal clinical measurement tool, such as FRAX™, which assesses 10-year fracture risk.

Target Audience:

HCPs specializing in endocrinology, internal medicine, and women’s health; physician assistants, nurse practitioners, and pharmacists who practice in those areas of specialty; and those who otherwise commonly care for or clinically encounter patients with postmenopausal osteoporosis.

By the end of the session the participant will be able to:

• Recall how the results of diagnostic imaging and pertinent past medical history to determine risk of fracture.
• List the criteria for initiation of pharmacotherapy for postmenopausal osteoporosis treatment and prevention and apply them to a patient case.
• Describe challenges associated with treating patients with postmenopausal osteoporosis, focusing specifically on the risks of the agents used to treat and prevent osteoporosis, and apply the information to optimize patient care in a patient case.
• Identify recently approved and emerging pharmacotherapeutic treatments for management of postmenopausal osteoporosis, and describe their mechanisms of action.

Hypoparathyroidism is a rare disorder characterized by decreased function of parathyroid glands resulting in low levels of parathyroid hormone (PTH). There are a variety of causes, including autoimmune disease, congenital defects, unintended parathyroid removal during thyroidectomy, or damage caused by radiation therapy. In a normal functioning parathyroid gland, PTH is secreted in response to low serum-ionized calcium. According to one study, nearly 60,000 people in the United States have been diagnosed with the disorder, with about 73% caused by surgery. Another study estimated the prevalence of primary hypoparathyroidism at 40 per 100,000 people in the United States.

Target Audience:

The following healthcare professionals: endocrinologists and primary care physicians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in endocrinology and internal medicine; and any other healthcare professionals with an interest in or who clinically encounter patients with hypoparathyroidism.

By the end of the session the participant will be able to:

• Describe the pathophysiology and manifestations of hypoparathyroidism and discuss how they may have relevance to treatment targets.
• Define patient-specific goals, identify treatments directly treating hypoparathyroidism, and incorporate both in the development of a treatment plan in patient cases.
• Describe goals and mainstays of supportive care in hypoparathyroidism and apply them to patient cases.
• Describe barriers to care in the optimal treatment of hypoparathyroidism and suggest strategies for ameliorating them.