Immune-Mediated Thrombocytopenia: A Clinical Overview (220)

Primary immune thrombocytopenia (ITP) is one of the most common hematologic conditions encountered in pediatric hematology and is a diagnosis of exclusion with no specific testing available to confirm the condition. While fairly consistent and typical in presentation, the astute clinician must be able to differentiate between immune and nonimmune etiologies of thrombocytopenia in order to confirm an accurate diagnosis, etiology, and treatment plan while shielding the patient from unnecessary testing and workup. Read more...

Compassionate Use, Expanded Access, and Patient Assistance ? Obtaining Life-Saving Medications When a Clinical Trial is Not Available (207-2)

The vast majority of clinical trials conducted in oncology are not open to patients under the age of 18 years old, leaving pediatric cancer patients with a very limited selection of FDA-approved therapies. Though clinical trials are the safest and most-beneficial means of determining the effectiveness of medications, not all patients have the option to enroll in clinical trials due to barriers such as patient clinical status, geographic location, or enrollment caps. In other circumstances where FDA-approved therapies are commercially available, insurance companies simply deny payment for off-label use of these expensive medications based on disease categories instead of oncogenic targets. Compassionate use programs, also known as expanded access trials or single patient investigational new drugs, provide opportunity for these patients to obtain possibly life-saving therapies. This interactive session will help demystify the process of gaining access to medical therapies on behalf of our patients. The different terms used in the compassionate use process will be discussed, as well as the specific steps oncology teams can take to apply to the FDA for approval of experimental medications. Case studies will provide examples of how the compassionate use process works in different circumstances as well as demonstrate clinical outcomes. Payment assistance programs and other resources to help patients pay for FDA-approved medications will also be covered. Read more...

CBD, THC, GVH, MMJ: Practical Ways to Make Sense of the Alphabet Soup (203)

Many pediatric oncology patients report medical marijuana (MMJ) and hemp-based CBD use. Hemp is now federally legal under the Farm Bill since December 2018 and hemp-based cannabinoid products are widely available. Research focused on cannabis and its’ use in oncology, hematology, and bone marrow transplant is continuing to grow. A study in Israel looks at adding cannabis to the treatment of graft versus host disease following bone marrow transplant. Read more...

What the HEC is MEC? Understanding Antiemetic Therapy for Moderately and Highly Emetogenic Chemotherapy (230)

Optimal chemotherapy induced nausea and vomiting (CINV) control has been shown to improve patient’s quality of life and decrease distress. Without good control, both physical and psychological complications can occur, including anorexia, malnutrition, and nutritional deficiency. But what is optimal CINV control? Nausea without vomiting? No nausea? No vomiting? Only vomiting a few times? This definition can mean something different to every patient, provider, and nurse. Read more...

Pediatric Anticoagulation: Who, What, When, Where, and Why (and a little How) (225) 

Pediatric anticoagulation can present challenges to the advanced practice nurse (as well as the ambulatory or bedside nurse) with limited experience in the broad scope of clinical scenarios where either prophylactic or treatment anticoagulation is recommended (it's not just clots). Traditionally, the only pharmacologic options utilized in children have been limited to heparin (including low molecular weight heparin) and warfarin. However, direct oral anticoagulants (DOACs) are now being incorporated in a limited fashion, dependent on both age of the child and clinical application. Read more...

Building Effective Partnerships in Nursing Practice and Research: Patient Advocacy Organizations and Nurses Supporting Each Other For Best Outcomes (227)

The engagement of patients and caregivers is increasingly more important in the design, conduct and dissemination of research (Kwon, Tandon, Islam, Riley, & Trinh-Shevrin, 2018). Patient advocacy organizations, such as Momcology, are often developed and led by patients, parents and/or caregivers. These organizations are one mechanism to consider when working to implement community engagement and recruitment strategies. Community engagement is far more than the addition of a community advisory board to organizations such as hospitals, agencies or research trials. Authentic engagement should mimic a partnership, including stakeholders in all phases of research from design, conduct, implementation, analysis and dissemination (Woolf, Zimmerman, Haley, & Krist, 2016). Read more...

Neurofibromatosis Therapeutics Program: Program Development, Tumor Treatment, and Side Effect Management (215)

The advanced practice provider is instrumental in designing, developing, and growing a program to treat tumors associated with Neurofibromatosis (NF) Type 1 and 2. Both NF1 and NF2 are autosomal dominant genetic disorders associated with the growth of both benign and malignant tumors of the peripheral and central nervous system. In the last two years selumetinib, a MEK inhibitor, gained FDA approval for treatment of plexiform neurofibromas in the children with NF. Read more...