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...
Speaker:
Alejandra Castillo MSN RN
CNE Hours
.50
8:30 am – 9:30 am Friday, September 16

General Session: State of Nursing: Pediatric Hematopoietic Stem Cell Transplantation: A Glimpse of the Past, Current State, a Glance of the Future (102)

Over the last 60 years, significant advances in in hematopoietic stem cell transplantation (HSCT) has provided improvements in effective treatment or cure and overall survival for many patients. There has been considerable HSCT progress and success in a variety of pediatric malignant and non-malignant diseases. This state of the science presentation will be providing the evolution and current and future highlights of Pediatric HSCT as it relates to disease conditions, stem cell sources, conditioning regimens, cellular immunotherapy, toxicity related treatments and supportive care. Read more...
2:45 pm – 3:15 pm Saturday, September 17

Renewed Hope for the Future: Menin Inhibitors for Relapsed/Refractory Leukemia (233)

Prognosis for pediatric patients and adolescents with multiple relapsed/refractory leukemia remains grim. Many have received relapsed protocols, CAR-T therapies, stem cell transplants and yet, the disease returns. In the past, the next discussion in this patient population would lead towards palliation and quality of life, with little hope for cure. Recent advancement in therapy for children with refractory leukemia has introduced the use of menin inhibitor in patients with KMT2A/MLL and NPM1 mutations, providing a number of young patients with another chance of remission and a chance at a cure. Read more...
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