Pediatric Blood and Marrow Transplant Consortium Educational Program
The Pediatric Blood and Marrow Transplant Consortium (PBMTC) Educational Program will offer a 1-day program focused on pediatric bone marrow transplant (BMT) preceding the ASPHO Annual Meeting. The sessions will invigorate the field of pediatric BMT, encourage physicians to learn more about a rapidly advancing field, and enable pediatric hematology/oncology practitioners who do a small amount of pediatric BMT to attend a meeting to which they normally wouldn’t have access.
To register for just the PBMTC program or for more meeting information, go to www.pbmtc.org.
Wednesday, April 26
(P01) Plenary Session I New Frontiers in Natural Killer Cell–Based Therapy
Mitchell S. Cairo, MD (Moderator); Michael Verneris, MD; Dean A. Lee, MD PhD
- Describe the latest development in ex-vivo expansion of NK cells and its potential application in relapsed Neuroblastoma.
- Report the safety and efficacy of haploidentical NK cells in combination with allogeneic stem cell transplantation in patients with relapsed/refractory AML.
- Summarize the pre-clinical effects of chimeric antigen receptor modified NL cells (NKCAR) in the treatment of B-cell NHL, Neuroblastoma and Sarcomas.
Natural Killer (NK) cells are a major component of human innate immunity. NK cells play an important role in tumor immunesurveillance and eradication and response to microbial infections. New therapeutic NK cell approaches are currently under investigation in a variety of pediatric malignancies. Pediatric hematopoietic stem cell transplant physicians, nurses, pharmacists and fellow have limited information regarding the potential of NK cell based therapy in pediatric cancer.
(P02) Plenary Session II Novel Approaches to Juvenile Myelomonocytic Leukemia (JMML)
Sung Choi, MD (Moderator); Franco Locatelli, MD PhD; Mignon Loh, MD
- Identify how diagnosis is made in JMML (criteria for establishing diagnosis).
- Describe standard treatment options, including allogeneic HCT (type of conditioning prep).
- Describe newer, novel therapies that are on the horizon (such as MEK inhibitor or hypomethylating agents).
Juvenile myelomonocytic leukemia (JMML) is a rare hematological malignancy occurring in approximately 2% to 3% of pediatric leukemia’s with a mean age at diagnosis of 2 years. It is classified as an overlap myelodysplastic syndrome/myeloproliferative disorder. Accordingly, there have been varying treatment approaches to JMML. Allogeneic hematopoietic cell transplantation (HCT) remains the only curative treatment for JMML. Although the exact cause of JMML remains unknown, mutations that cause JMML have been linked to a variety of genes: RAS family of oncogenes, NF1, PTPN11, or CBL. Newer targeted therapies are being explored based on novel insights gained from the laboratory.
10:45 am–12:25 pm
(P03) Plenary Session III Advances in Stem Cell Collection and Transfusion Medicine
Angela R. Smith, MD MS (Moderator); Paul Carpenter, MD; Stefan Ciurea, MD; Rachel S. Bercovitz, MD MS; Sonata Jodele, MD
- Describe the best practices for peripheral blood stem cell collection.
- Identify new techniques for peripheral blood stem cell collection.
- Recognize the impact of anti-HLA antibodies on graft failure.
Peripheral blood stem cell collection practices vary significantly among institutions. Many institutions have developed algorithms, but optimal regimens have not been defined. Additionally, donor specific anti-HLA antibodies can impact the success of stem cell transplant. The importance of these antibodies is now being recognized. By the end of this session, participants should be able to describe best practices for peripheral blood stem cell collection, identify new techniques for peripheral blood stem cell collection and recognize the impact of anti-HLA antibodies on graft failure.
(P04) Workshop I Complications in Bone Marrow Transplantation: How Do I Treat?
Julie An Talano, MD (Moderator); Nancy Kernan, MD
- Describe the best practices for diagnosis and treatment of TMA.
- Describe the best practices for diagnosis management and treatment of veno-occlusive disease of the liver.
The ability to reduce transplant-related morbidity and mortality by better care of two major complications is the focus of this session: “Complications in Bone Marrow Transplantation: How do I Treat”. Leading experts will review the latest diagnostic criteria and treatments for two rare but challenging complications of SCT - Veno Occlusive Disease (VOD) of the Liver and thrombotic microangiopathy (TMA). There are recently published reports of promising treatments for these disorders in SCT patients which may translate into improved survival.
(P05) Plenary Session IV Unique Challenges in HSCT for Rare Nonmalignant Diseases
Andrew C. Dietz, MD MS (Moderator); Elizabeth Kang, MD; Lauri M. Burroughs, MD
- Identify rare diseases (DC, CGD, and IPEX) that require unique needs specific to their disease during HSCT.
- Develop treatment plans for successful HSCT outcomes in rare diseases (DC, CGD and IPEX).
- Modify care practices for patients with DC, CGD and IPEX.
Particular attention is required when designing hematopoietic stem cell transplantation (HSCT) conditioning regimens and supportive care practices for patients with rare nonmalignant diseases. HSCT differs significantly for patients with Dyskeratosis Congenita (DC), Chronic Granulomatous Disease (CGD), and Immune dysregulation Polyendocrinopathy Enteropathy X-linked syndrome (IPEX) due to underlying pre-transplant genetic and medical factors. Recent published reports for each of these diseases outline unique approaches. By the end of this session, participants will be able to identify rare diseases that need special attention, develop the most up-to-date plans for successful HSCT, and be able to modify care practices for these patients appropriately.
(P06) Nursing Plenary Session
Rita Secola, PhD RN FAAN CPON® (Moderator); David Margolies, MD
(P07) Oral Abstract Presentations
Reception and Poster Session
Evening Dinner and Young Investigator Workshop—Speed Mentoring
Moderators: Julie An Talano, MD; Dean Lee, MD PhD
No Relevant financial relationships to disclose: Rachel Bercovitz; Lauri Burroughs; Paul Carpenter; Sung Choi; Andrew C. Dietz; Elizabeth Kang; Nancy Kernan; Mignon Loh; Angela R. Smith, Julie An Talano
Relevant financial relationships to disclose and no discussion of off-label drug use: Mitchel S. Cairo- Jazz Pharmaceuticals/Gilead Celgene/Roche: Advisory Board Member, Honoraria; Sanofi: Speaker Bureau, Honoraria; Stefan Ciurea- Cyto-Sen: Founder, stock; Spectrum: Advisory board member, honoraria; Dean Lee-Intrexon: Intellectual property license, Stock; Ziopharm: Intellectual property license, Stock; Ziopharm/Intellia Therapeutics/Jazz Pharmaceuticals: Consultant, Consultant fee; Shire US: Advisory Board Member, honorarium; Courier Therapeutics: Advisory Board Member, honorarium and stock options; Cyto-Sen Therapeutics: Founder, Ownership Interest; Medac Pharma: Advisory Board Member, travel reimbursement; Michael Verneris- Biogen: Advisory, stock
Relevant financial relationships to disclose; discussion of off-label drug use: Sonata Jodele- Alexion: Lecture, Honoraria
Julie An Talano, MD, Chair
Children’s Hospital of Wisconsin
Mitchell S. Cairo, MD
New York Medical College
Sung Choi, MD
University of Michigan
Ann Arbor, MI
Andrew C. Dietz, MD MS
Children’s Hospital Los Angeles, University of Southern California
Los Angeles, CA
Michael Pulsipher, MD
Children’s Hospital Los Angeles, University of Southern California
Los Angeles, CA
Rita Secola, PhD RN FAAN CPON®
University of Southern California School of Nursing
Los Angeles, CA
Angela R. Smith, MD MS
University of Minnesota
No Relevant financial relationships to disclose: Sung Choi; Andrew C. Dietz; Michael Pulsipher; Rita Secola; Angela R. Smith; Julie An Talano
Relevant financial relationships to disclose: Mitchell S. Cairo- Jazz Pharmaceuticals/Gilead/Celgene/Roche: Advisory board member, honoraria; Sanofi: Speaker bureau, honoraria
Accreditation Subcommittee Disclosures
No relevant financial relationships: Daniel S. Wechsler; Jacqueline Baskin; Leo Mascarenhas, Tanya Brown
Continuing Medical Education Credit
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of the American Society of Pediatric Hematology/Oncology and Pediatric Blood and Marrow Transplant Consortium (PBMTC). The American Society of Pediatric Hematology/Oncology is accredited by the ACCME to provide continuing medical education for physicians. The American Society of Pediatric Hematology/Oncology designates this live activity for a maximum of 8 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
ASPHO requires that planners and presenters disclose the existence of any relevant financial or other relationship they and/or their spouse/partner have with the manufacturers of any commercial interest (defined as any entity producing, marketing, reselling, or distributing healthcare goods or services consumed by, or used on, patients) whose products or services relate to the topics presented. ASPHO also requires disclosure of the intent to discuss unlabeled or investigational use(s) of a commercial product. The Certification and Continuing Education Committee reviews potential conflicts of interest as submitted by planners and presenters and resolves such conflicts so as to ensure the content of the activity is aligned with the interests of the public.
The material presented in this activity represents the opinion of the speakers and not necessarily the views of ASPHO or PBMTC.