10:15 am – 10:45 am Friday, September 16

HSCT for MPS disorders (Hunters and Hurlers), (217-2)

Hematopoietic Stem Cell Transplant (HSCT) is best known as a treatment modality for hematologic malignancies, some solid tumors, and various autoimmune disorders. It is lesser known as a means to halt progressive symptoms in certain chromosomal inherited disorders such as Hunters and Hurlers disease. Patients with Hunters and Hurlers are born with an inherited x-linked chromosomal disease where sugar molecules are unable to be broken down and digested by the body causing both physical and mental disabilities. There is no cure for these diseases but HSCT is an intensive therapy that has shown promise with these diagnoses, helping to stop progression of symptoms of disease.

Two case studies of children who underwent HSCT at our center, one for Hunter’s and one for Hurler’s, will be presented from both a medical and psychosocial standpoint. How these children first presented, how HSCT affect their disease, the complications they underwent during their HSCT, and the complex psychosocial aspects of receiving chemotherapy as well as an extended hospitalization.

Learning Objectives:

  • The learner will describe the role of Hematopoietic Stem Cell Transplant in the treatment of non-malignant diseases.
  • Identify conditioning regimens and their unique complications for non – malignant diseases.
  • The learner will describe the psychosocial needs of hematopoietic transplant patients and their families when they undergo a complex regiment such as HSCT.
4:15 pm – 5:15 pm Friday, September 16

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...
5:00 pm – 6:00 pm Thursday, September 15

Toward a Cure for Sickle Cell Disease: Exploring Gene Therapy (210)

Sickle cell disease (SCD) is the most common inherited blood disorder worldwide. It is estimated that 300,000 babies are born with SCD every year and it occurs largely in descendants of Sub Saharan Africa. SCD is caused by a specific mutation in the beta globin gene that results in sickle hemoglobin production. Sickle hemoglobin polymerization leads to red blood cell sickling, chronic hemolysis and vaso - occlusion. Patients with SCD experience significant pain crisis and end organ damage that leads to a decreased lifespan with a median life expectancy in the United States of 43 years. At this time, the only known cure for SCD is hematopoietic stem cell transplantation (HSCT) most often with matched sibling donors. Read more...
5:00 pm – 5:30 pm Thursday, September 15

The Role of the Pediatric Clinical Research Nurse in the Management of Severe Aplastic Anemia (208-1)

Severe aplastic anemia (SAA) is a serious and life-threatening condition with an unknown etiology, involving improper production of stem cells due to damaged bone marrow. Without functional stem cells, the body is depleted of red blood cells, white blood cells, and/or platelets. With only 600 to 900 cases diagnosed each year in the U.S. alone, SAA is rare. Most patients are only provided supportive care measures: receiving blood transfusions to manage symptoms and adhere to complex medication regimens to limit complications; however, advancements in treatment of SAA are made possible through clinical research trials. The Clinical Research Nurse (CRN) must address the clinical needs of the research patient, but also be sensitive to the psychosocial and ethical issues of supporting pediatric research patients. This case study presents a 7-year old Mongolian patient, “M.B.”, with refractory SAA, who failed standard immunosuppressive therapy and failed to engraft from a previous expanded umbilical cord stem cell transplant. M.B. was enrolled in a clinical research protocol in which he underwent a haplo-identical transplantation using peripheral blood stem cells and post-transplant GVHD prophylaxis using Cyclophosphamide. M.B.’s complex clinical trajectory will unfold over the course of the case study, with the role of the Clinical Research Nurse highlighted. In addition to clinical complications, the COVID-19 pandemic added an additional layer of psychosocial and ethical complexities for M.B. and his family to navigate. Application of the Clinical Research Nurse Domain of Practice ensures that the bedside nurse addresses the holistic needs of the research patient and caregiver at the bedside, while also maintaining the integrity of the research protocol. Read more...
Alejandra Castillo MSN RN
CNE Hours
3:00 pm – 4:00 pm Friday, September 16

General Session: Sickle Cell Disease: Pathophysiology, Pain, and Prevention of Problems (103)

Sickle Cell Disease (SCD) is the most common inherited hemoglobinopathy in the United States; the etiology is a DNA mutation that results in the different amino acids at position 6 of the beta-globin chain, where valine is produced instead of glutamine. The sickling of the red blood cell sets off a cascade of pathology. Vaso occlusion causing ischemia to almost every body system accounts for the majority of morbidity and is the etiology of acute pain episodes. Acute pain episodes account for the majority of health care utilization by this population.

2:15 pm – 3:15 pm Saturday, September 17

Strategies in Survivorship Care: Addressing Current Long-term Follow-up Surveillance Gaps Through Individualized Survivorship Care Plans And Specialty Survivorship Clinics (234) 

With current 10-year survival rates greater than 80% for pediatric, adolescent, and young adult (AYA) cancer patients there exists and ever-increasing population of pediatric and AYA cancer survivors. Pediatric and AYA survivors who finish cancer-directed treatment are often burdened with significant risks for long-term complications. This includes risks of secondary cancers and accelerated development of usual age-related comorbid conditions such as heart failure, kidney disease and osteopenia. Given these risks, cancer survivors require specialized health care monitoring and surveillance. The Childhood Cancer Survivor Study has identified significant, suboptimal adherence to COG screening guidelines for secondary malignancies (breast, colorectal, and skin) and cardiac disease amongst this high-risk population. Many survivors and their caregivers also report that they feel uninformed yet worried about potential late effects. This current gap emphasizes the importance of individualized survivorship care post cancer treatment, both short and long term.

Stephanie Neerings MSN APRN FNP-BC
CNE Hours
10:30 AM - 11:30 AM Saturday, September 17

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...
9:45 am – 10:15 am Friday, September 16

Sickle Cell Disease: When Abdominal Pain is Not Sickle Cell Pain (213-1)

A hallmark of sickle cell disease is pain. Abdominal pain can be a common complaint among children with and without sickle disease and may be related to a wide variety of underlying causes For children with and without sickle cell disease abdominal pain may be related to constipation, anxiety, an infectious process, an acute surgical concern or possibly a chronic medical condition. Abdominal pain in a child with sickle cell disease may be related to any of the previously mentioned causes but may also be related to splenomegaly, hepatomegaly, cholelithiasis, or a vaso-occlusive crises. Read more...
10:15 am – 10:45 am Friday, September 16

Sickle Cell Disease and Increased Hemolysis. When to Consider Evaluating for A Concurrent Hereditary Hemolytic Condition (213-2)

Sickle Cell Disease is an inherited red blood cell disorder that can lead to increased hemolysis which can result in significant anemia. When a child with sickle cell disease does not respond to therapies or interventions designed to decrease hemolysis, then a provider may want to consider the presence of a concurrent inherited hemolytic process such as G6PD, Hereditary Elliptocytosis or Gluthathione reductase deficiency. Read more...
9:45 am – 10:50 am Friday, September 16

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...
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