Free Pediatrics CME

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.

Hemophilia is a genetic disease caused by mutation of one of the genes for coagulation proteins leading to dangerous, uncontrolled bleeding. In hemophilia B, a mutation in the gene for factor IX (FIX) leads to an endogenous deficiency in the clotting factor. The incidence of hemophilia B is the same in all geographic regions, populations, and ethnic groups,affecting approximately 1 out of every 30,000 male births. The condition is diagnosed by measuring FIX activity, and patients with severe hemophilia have levels of 1% or less.

Patients with severe hemophilia B are at risk for spontaneous, life-threatening bleeding episodes. Untreated, the life expectancy is approximately 20 years,and painful or even life-threatening morbidities include intracranial hemorrhage, severe bleeding in other organ systems, musculoskeletal injury, and joint injury. In contrast, in people with moderate or mild hemophilia, abnormal bleeding usually occurs after minor trauma or surgery.

Physical therapy can ease symptoms if internal bleeding has damaged a patient’s joints, and surgery may be necessary if internal bleeding has caused severe damage. However, the current standard of therapy for hemophilia B is intravenous infusion of therapeutic factor concentrates. Through the reduction in the number of bleeding incidences and improvement in quality of life, factor replacement therapy has significantly reduced the morbidity and mortality of hemophilia. Furthermore, prophylactic therapy has the demonstrated benefit of reducing the development of hemophilic arthropathy.

Target Audience:

The following healthcare professionals: hematology, primary care physicians, and pediatricians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in hematology as well as other Hemophilia Treatment Center HCPs; and any other clinicians with an interest in hemophilia B.

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

• Describe the risk factors and occurrence of hemophilia B.
• Identify available prophylactic and treatment options for hemophilia B and apply them to a patient case.
• Identify the new treatment options for hemophilia B.
• Identify adherence barriers in and deliver effective treatment counseling to patients with hemophilia B.

Hemophilia is a genetic disease caused by mutation of one of the genes for coagulation proteins leading to dangerous, uncontrolled bleeding. In hemophilia A, a mutation in the gene for factor VIII (FVIII) leads to an endogenous deficiency in the clotting factor. The incidence of hemophilia A is the same in all geographic regions, populations, and ethnic groups, affecting approximately 1 out of every 5000 male births. The condition is diagnosed by measuring FVIII activity, and patients with severe hemophilia have FVIII activity of 1% or less. Patients with severe hemophilia A are at risk for spontaneous, life-threatening bleeding episodes. Untreated, the life expectancy is approximately 20 years, and painful or even life-threatening morbidities include intracranial hemorrhage, severe bleeding in other organ systems, musculoskeletal injury, and joint injury. In contrast, in people with moderate or mild hemophilia, abnormal bleeding usually occurs after minor trauma or surgery.

Target Audience:

The following healthcare professionals: hematology, primary care physicians, and pediatricians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in hematology as well as other Hemophilia Treatment Center HCPs; and any other clinicians with an interest in hemophilia A.

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

• Describe the risk factors and occurrence of hemophilia A.
• Identify available prophylactic and treatment options for hemophilia A and apply them to a patient case.
• Identify the new treatment options for hemophilia A.
• Identify adherence barriers in and deliver effective treatment counseling to patients with hemophilia A.

Activity Description / Statement of Need:

In this online, self-learning activity:

Crohn’s disease (CD) is an inflammatory bowel disease (IBD) that is defined by a transmural process that often occurs in the terminal ileum but may occur in any portion of the GI tract. Although the exact etiology of CD is unknown, a handful of genetic, immunological, and environmental risk factors have been identified, including an impaired immune response to commensal or pathogenic intestinal microbiota that drives mucosal inflammation in patients who are genetically susceptible.

Intestinal and abdominal complications such as strictures, abscesses, and fistulas are common among pediatric patients and increase as the disease progresses. Vitamin and mineral deficiencies have also been attributed to IBD due to mucosal inflammation in the gut, low oral intake, and malnutrition resulting in complications such as poor bone health, delayed puberty, anemia, and stunted growth. The annual direct healthcare costs – accounting for the majority of costs in the U.S. – are about $18,000 to $19,000 per patient. CD shares a number of clinical characteristics with other disease states, and initial misdiagnosis and delayed diagnosis of pCD are not uncommon, which may have a dramatic impact on a patient’s clinical course because pCD is often more severe and associated with a higher incidence of complications than adult CD.

This learning activity has been designed to bring healthcare professionals’ knowledge of the strategies to manage pCD up to date and to improve their competence and performance in treating it.

Target Audience:

The following healthcare professionals: pediatricians, pediatric gastroenterologists, and those who specialize in adolescent medicine; physician assistants, nurse practitioners, and pharmacists; and any other healthcare professionals with an interest in or who clinically encounter patients with pCD.

Activity Description / Statement of Need:

In this online, self-learning activity:

The WHO defines vaccine hesitancy (VH) as a “delay in acceptance or refusal of vaccines despite the availability of vaccination services.” Despite substantial progress in rates of routine immunization over the decades prior to the most recent one, more recent trends suggest that immunization rates are beginning to plateau. Beyond VH and skepticism, there is also the embrace of outright vaccine rejection or denial fostered by the presence disinformation on conventional and social media platforms including claims that vacines are unsafe or unnecessary. Recent outbreaks of largely eradicated diseases such as measles, mumps, and diphtheria suggest that herd immunity may have suffered, putting those ineligible for vaccination at additional risk of infection. These developments have been attributed in part to VH and denial.

One large group with increasing VH is parents. A 2019 national survey found that approximately 1 in 4 parents reported serious concerns towards vaccinating their children. Another study saw that in up to 35% parents of well-vaccinated children demonstrate VH. Parents may raise issues that many providers feel ill-equipped to answer, due to lack of thorough knowledge of all vaccines or lack of evidence-based communication strategies. Unfortunately, only few evidence-based strategies exist to guide providers in their discussions with vaccine-hesitant parents.

Providers play a crucial role in vaccinating populations, but it is not and should not be their sole responsibility. Clinical practice sites, community organizations, health organizations, and government all contribute to addressing VH. Understanding potential solutions outside the office, such as media campaigns and policy changes, also provide insight into vaccine hesitancy and potential directions for future use.

Target Audience:

The following HCPs: Primary care physicians, pediatricians, and public health professionals; physician assistants, nurse practitioners, nurses, and pharmacists who practice in adult internal medicine and pediatrics; and any other clinicians who commonly encounter patients eligible for protection against vaccine-preventable diseases.

Activity Description / Statement of Need:

In this online, self-learning activity:

Hemophilia is a genetic disease caused by mutation of one of the genes for coagulation proteins leading to dangerous, uncontrolled bleeding. In hemophilia B, a mutation in the gene for factor IX (FIX) leads to an endogenous deficiency in the clotting factor. The incidence of hemophilia B is the same in all geographic regions, populations, and ethnic groups, affecting approximately 1 out of every 30,000 male births. The condition is diagnosed by measuring FIX activity, and patients with severe hemophilia have levels of 1% or less. Patients with severe hemophilia B are at risk for spontaneous, life-threatening bleeding episodes. Untreated, the life expectancy is approximately 20 years, and painful or even life-threatening morbidities include: intracranial hemorrhage, severe bleeding in other organ systems, musculoskeletal injury, and joint injury. In contrast, in people with moderate or mild hemophilia, abnormal bleeding usually occurs after minor trauma or surgery. Unfortunately, the literature shows that not only do clinicians struggle with the classification of hemophilia severity and that there are gaps in knowledge present that contribute to delayed diagnosis and treatment, with an attendant increase in morbidity and mortality. Challenges in diagnosis and classification are only the first of several gaps in care that patients with hemophilia face.

Target Audience:

The following HCPs: hematologists and pediatricians; physician assistants, nurse practitioners, and pharmacists who practice in hematology, and other HCPs who practice in hemophilia treatment center; and any other clinicians with an interest in or who clinically encounter patients with hemophilia B.

Activity Description / Statement of Need:

In this online, self-learning activity:

Pompe disease (PD) is a progressive, often fatal, autosomal recessive, neuromuscular disorder caused by mutations of the α-glucosidase gene on chromosome 17. PD is characterized by glycogen accumulation in skeletal, cardiac and smooth muscles due to a deficiency in α-glucosidase (GAA), an important lysosomal enzyme responsible for glycogen catabolism. PD is categorized into three groups based on symptoms and age of onset. The classic infantile form presents in the first year of life, usually in the first two months, with hypertrophic cardiomyopathy. The non-classic infantile form presents later in the first year of life, without or with less severe cardiomyopathy. The late onset form of PD presents any time after one year of life, usually without cardiac complications.

PD is rare, with one study estimating the incidence in the U.S. to be 1 in 22,000 births. The biggest risk factor for the disease is genetics; at conception, each sibling of an affected individual has a 25% chance of being affected, a 50% chance of being an asymptomatic carrier, and a 25% chance of being not a carrier. Nonspecific diagnostic findings for PD may include abnormal EMG, elevated serum creatine kinase, elevated transaminases and elevated lactate dehydrogenase. A definitive diagnosis for PD involves a dried blood spot test to determine GAA enzyme activity level; reduced GAA activity (less than 40% of normal) is indicative of a positive diagnosis. A gene mutation analysis, or another GAA enzyme test using a different area of tissue, is recommended to confirm the diagnosis. The disease is not uncommonly undiagnosed or is misdiagnosed, representing one practice gap that continued HCP education may address, particularly given that earlier treatment may minimize rapid and irreversible disease progression.

Target Audience:

The following HCPs: Neurologists, pediatricians, and primary care physicians; physician assistants and nurse practitioners in those areas of specialty; pharmacists who practice in specialty pharmacies that treat patients with rare diseases; and any other HCPs with an interest in or who may clinically encounter patients with PD.

Activity Description / Statement of Need:

In this online, self-learning activity:

Mucopolysaccharidoses (MPS) are a group of genetic diseases characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), resulting in a variety of clinical manifestations in patients presenting with MPS. MPS has several subcategories, and MPS type I (MPS I) arises from the inheritance of an alteration in the IDUA gene, which encodes for alpha-L-iduronidase. Affecting an estimated one in 100,000 live births, MPS I is categorized as either attenuated MPS I (also known as Scheie or Hurler-Scheie syndromes) or severe MPS I with cognitive impairment (also known as Hurler syndrome).

Progressive in nature, MPS I is associated with multi-organ complications and sequelae. Patients exhibit a spectrum of clinical presentations, including facial deformities, organomegaly, cognitive impairments, upper airway obstructions, skeletal deformities, and cardiomyopathy. The burden of MPS I is considerable, with reports of caregivers contributing 51 hours per week on average to help patients perform daily activities of living. Quality of life for patients and their caregivers is significantly reduced with MPS I, affecting the social, emotional, and financial well-being of a family. It is reported that parents fear for their child’s delayed language acquisition, ability to fit in amongst peers and the society, fear of the expense for the necessary care, and fear for the death of a child from obstructive sleep apnea.

This learning activity has been designed to bring HCPs’ knowledge of the strategies for treatment and management of MPS I up to date and to improve their competence and performance in treating it.

Target Audience:

The following HCPs: Pediatricians, neurologists, endocrinologists, genetic disease specialists; physician assistants, nurse practitioners, and pharmacists in the aforementioned areas of specialty; and any other HCPs with an interest in or who may clinically encounter patients with MPS I.

Activity Description / Statement of Need:

In this online, self-learning activity:

Advances in ALL management has led to considerable improvements in outcomes over the past several years, with modern-day treatment leading to remission rates of up to 99% and cure rates of about 90% in children with ALL. However, achieving these high rates of response require multiple phases of treatment, including induction, consolidation, interim maintenance, delayed intensification, and maintenance therapy. 

Chemotherapy was previously the standard of care in this setting but is associated with considerable toxicities. Novel, less toxic options are available or being studied for children who have relapsed after treatment as are new pharmacotherapies specific to patients with Philadelphia chromosome–positive disease and novel formulations of asparaginase. Helping the clinician discern the role of each of the first- and subsequent-line novel therapies based on the most up-to-date research merits continuing education programming in ALL. Indeed, a survey of oncology providers revealed that 86% indicated that added professional guidance would be helpful in incorporating more recent therapies into care.

And despite the inclusion of newer agents in pediatric ALL guidelines, a survey of oncologists and other providers who treat patients with the condition found that 40% of respondents had never prescribed, dispensed, or administered them. Challenges include lack of expertise and management of side effects. Furthermore, recently published guidelines, therefore, also inherently suggest a gap in medical practice and justify the need for educational programming.

Target Audience:

HCPs specializing in: pediatric hematology-oncology, hematology, oncology, pathology, and those who otherwise commonly care for or clinically encounter pediatric patients with ALL.

Activity Description / Statement of Need:

In this online, self-learning activity:

Atopic dermatitis (AD) is a chronic, highly pruritic inflammatory skin disease that is one of the most common skin disorders in children but may develop at any age. It affects 15-30% of children and two to ten percent of adults in developed countries, and between 10-30% of children who have the condition continue to experience it in adulthood. AD is thought to arise from a complicated interplay between multiple genes and environmental triggers, with known risk factors including family history and loss of function mutations in filaggrin. Complications include food allergy, asthma, and allergic rhinitis, and aside from genetics, its pathophysiology involves T-cell mediated inflammation and epidermal dysfunction. The disease is associated with a considerable healthcare burden placed on patients and their families; pruritis aside, patients not uncommonly suffer a loss of sleep and experience secondary infections, anxiety, and depression.

Target Audience:

The following HCPs: Pediatric and adult dermatologists, allergists, and internists; physician assistants, nurse practitioners, and pharmacists who treat patients with dermatologic conditions; and any other HCPs with an interest in or who diagnose, treat, or manage patients with AD.