Objectives:

1. Improve the pediatric hematologist/oncologists understanding of the classification of vascular anomalies.
2. Provide an understanding of treatment options for vascular tumors and malformations.
3. Provide an understanding of associated syndromes and genetic discovery to improve clinical acumen and improve the understanding of the use of surgery and interventional radiology in the treatment of vascular anomalies.
4. Develop an understanding of the pathological differences of varied vascular anomalies.

The Vascular Anomalies Special Interest Group was started in 2013, and consistently has seen participation by 10%–15% of annual meeting registrants. This field historically has been dominated by surgeons, interventional radiologists, and dermatologists. Because of recent successes in medical therapies led by pediatric hematology/oncology providers and the implementation of collaborative research programs, however, the role of the pediatric hematologist/oncologist in this field has become clear. There is an acute need for our community to acquire basic knowledge about the diagnosis and management of these conditions.

Advanced registration is recommended. The registration fee is $25.

Supported in part by vascular anomalies patient support groups.

The meeting will provide the Research, Practice, and Education Committees’ ongoing studies and brainstorm for new projects. Advanced registration is recommended.

Big Lessons from a Small Disease: “Stumbling from failure to failure with no loss of enthusiasm."

Objectives:

1. Discuss the roles of mentorship and collaboration in forging a career.
2. Relate that the importance of a rare disease is often inversely proportional to its incidence.

Diamond Blackfan anemia is a very rare inherited bone marrow failure syndrome; incidence 5 -10 per million live births. The disorder, first described in 1936, is characterized in classic cases by erythroid failure resulting in severe anemia, birth defects and a marked predisposition to cancer. The majority of genetically defined cases result from mutations or deletions in one of more than 15 genes encoding either a small or large subunit – associated ribosomal protein resulting in haploinsufficiency of that ribosomal protein. The somewhat restricted phenotype and the predisposition to cancer resulting from a defect in translation are both intriguing and unexplained. This talk will review clinical and laboratory research over the past 40 years that has led to our current understanding of this disorder. Emphasis will be placed on the influence of mentorship and collaboration in the discovery upon which our current knowledge is based.

No relevant financial relationships and no discussion of off-label drug use.

Objectives:

1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
2. Examine current research outcomes from a variety of the latest in basic and clinical research.

The 2017 Program Committee has chosen the abstracts listed below as among the best submissions.

Transgenic Expression of IL15 Improves Antiglioma Activity of IL13Rα2-CAR T Cells (4001)
Giedre Krenciute, PhD

P2Y12 Receptor Function and In Vivo Platelet Response to Cangrelor in Neonates with Cyanotic Congenital Heart Disease (4002)
Thomas Diacovo, MD

No relevant financial relationships and no discussion of off-label drug use: Giedre Krenciute

Relevant financial relationships and discussion of off-label drug use: Thomas Diacovo - The Medicine Compnay; Principle Investigator, research grant

Objectives:

1. Identify cancers and other features associated with predisposition syndromes and the genes involved in predisposition syndromes.
2. Discuss the unique aspects of genetic counseling for children and families with cancer predisposition syndromes (as compared to adults).
3. Develop tumor surveillance strategies for children with cancer predisposition syndromes.

Cancer predisposition syndromes are being diagnosed at increasing rates and new ones continue to be discovered. However, optimal counseling, testing, and management strategies are undefined. Pediatric oncologists should be knowledgeable about the presentation and genetics of cancer predisposition syndromes and the unique aspects of genetic counseling for children and families with cancer predisposition syndromes (as compared with adults) and be able to develop tumor surveillance strategies for children with cancer predisposition syndromes.

Objectives:

1. Discuss challenges in design and implementation of pediatric thrombosis clinical trials.
2. Recognize areas of active investigation in pediatric thrombosis and understand the data supporting studies compared to standard of care.

Despite the increasing attention to thrombosis in children, most evidence guiding pediatric practice is taken from adult data and clinical experience. In recent years, randomized controlled trials dedicated to pediatric patients have begun to address major questions in thrombosis. This session will highlight areas of equipoise by reviewing these planned and open trials. Current studies are addressing length of anticoagulant therapy, prophylaxis in leukemia, catheter-related thrombosis, and thrombolysis versus anticoagulation for proximal deep vein thrombosis.

Objectives:

1. Explore why interprofessional team development is important in patient care and the future of healthcare.
2. Choose the elements of a high-functioning interprofessional team.
3. Sketch the care of a complex hematology-oncology patient using an interprofessional team approach.
4. Chart the structure of an interprofessional team at your institution.

The care of pediatric hematology-oncology patients requires a diverse set of healthcare professionals. Interprofessional collaboration between these individuals improves coordination of care, communication, and patient safety. However, the how- to of interprofessional team development is only infrequently incorporated into health professional training programs. In this session, we will introduce tools for interprofessional team development and allow participants to practice collaborative communication using real-world scenarios. This experience will encourage using interprofessional teams in daily practice.

No relevant financial relationships to disclose; discussion of off-label drug use: Brooke Bernhardt

No relevant financial relationships to disclose and no discussion of off-label drug use: Kathleen Adlard

Relevant financial relationships to disclose and no discussion of off-label drug use: Richard Tower- Abbott Labs: Family-employee, salary

Speed mentoring is a series of short, focused conversations with a mentor about specific questions. It is a relatively new session concept that focuses on quick-hit information for time- efficient networking. Fellows and recent graduates registering for this Speed Mentoring session will have the opportunity to meet one-on-one with up to three mentors for 10 minutes each. Mentees will be contacted before the session and will be required to submit a one-page document prior to arriving in Montréal that addresses the focus of the speed mentoring session’s topic of interest (e.g., CV; abstract; specific aims of grant proposal; or preliminary data in the form of table, figure, poster draft, or design, etc.). Mentees should come prepared with two to three concrete questions to discuss for their chosen subject. Mentors will review mentees’ documents ahead of time for meaningful insight and efficient engagement. Mentees will be randomly assigned to one of two sessions taking place at 4:45–5:20 pm and 5:30–6:05 pm.

Advanced registration is required.

The mission of the GSIG is to improve the diagnosis, care, and outcomes for children with hematologic and oncologic disease across the world through the development of training partnerships and collaborative research. Advanced registration is recommended.

Dr. Isaac Odame will discuss how lack of diversity of pediatric hematologists/oncologists have contributed to the lack of hematopoietic stem cell transplants (HSCT) performed to cure pediatric patients with sickle cell disease and other healthcare disparities. With this foundational knowledge, workshop attendees will further develop this year’s workshop theme during the “Open Space” interactive networking sessions that follow. Funding for the Diversity SIG workshop is provided by a grant (R13CA213938) from the National Cancer Institute. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services, nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. government. The DSIG provides a national networking forum for members who are interested in promoting diversity in the field of pediatric hematology/oncology. The DSIG also aims to support the advancement of all ASPHO members and the field by broadly increasing diversity and inclusiveness in the membership and leadership of ASPHO, as well as in the home institutions of members.

Advanced registration is recommended.

ASPHO is holding its fourth annual 5K Fun Run/Walk during the annual meeting at held at the Old Port of Montreal. Registration is $25 prior to April 27; race-day registration is $35. Registrants will receive a T-shirt. You can register when you register for the meeting. Exhibitors interested in participating, please contact This email address is being protected from spambots. You need JavaScript enabled to view it. .

Objectives:

1. Enhance the understanding and improve utilization of the concept of Concurrent Care.
2. Identify strategies for identifying and referring patients to hospice and palliative care services as well as barriers to those referrals.
3. Discuss patient and family personal experiences with hospice and palliative care services.

Recognition of the importance of pediatric palliative care has grown; however, a gap still exists in the consistent delivery of multidisciplinary palliative care to patients receiving disease- directed therapy. With the Affordable Care Act, pediatric palliative and hospice care can be provided along with concurrent disease-directed therapy, but providers’ lack of understanding of these services leads to inconsistent referral practices and confusion. This session addresses some of the common misunderstandings and aims to improve quality of care across the continuum.

Objectives:

1. Discuss recent research findings relevant to inherited platelet disorders, including their history, clinicopathologic features, suggested diagnostic and therapeutic approaches and molecular basis.
2. Describe capabilities and limitations of currently available methods for platelet function testing and select a suitable diagnostic algorithm for suspected platelet dysfunction in children.
3. Select appropriate antiplatelet agents and consequent dosing and laboratory monitoring options in pediatric patients.

Recent research has identified the molecular basis for several inherited platelet disorders and confirmed the utility of various new antiplatelet agents. However, the low frequency of these disorders and the challenges of platelet function testing contribute to a gap between contemporary research findings and clinical practice. Clinicians must possess a working knowledge of recent developments in platelet disorders, platelet function testing, and antiplatelet agents to optimize patient care within this niche arena of pediatric hematology.

Objectives:

1. Discuss the impact of MRD status both pre- and post- SCT in patients with T-cell ALL.
2. Describe the indications of allogeneic SCT for patients with Ph+ ALL in the era of BCR-ABL tyrosine kinase inhibitors.
3. Identify appropriate candidates for immunotherapy pre- or post- SCT in pediatric patients with B-cell ALL. Allogeneic SCT can effectively treat patients with refractory/ relapsed ALL.

Allongeneic SCT can effectively treat patients with refractory/relapsed ALL. However, transplant-related morbidity/mortality and post-HSCT relapse remain significant limitations. With the advent of improved predictors of outcomes (e.g., MRD status) and innovative therapies such as targeted, biologically- relevant agents (e.g., BCR-ABL tyrosine kinase inhibitors) and immunotherapies (e.g., bispecfic T-cell engagers and CAR-T cell therapy), the indications and timing of HSCT for this patient population are evolving rapidly. Thus, pediatric hematology/ oncology providers need to understand how these novel therapeutic approaches and “MRD status” results are being integrated into SCT-based regimens.

St. Baldrick’s Foundation Robert J. Arceci Innovation Award Announcement

Frank A. Oski Memorial Lectureship

Alexis Thompson, MD MPH
Advances in the Hemoglobinopathies
Objectives:

1. Review recent and potentially practice-changing clinical studies in sickle cell disease and thalassemia.
2. Explore emerging opportunities to improve clinical care and advocacy in non-malignant hematology.

Dr. Alexis Thompson is currently the Hematology Section Head and holds the Sarah and A. Watson Armour Chai in Childhood Cancer and Blood Diseases at the Ann and Robert H. Lurie Children's Hospital of Chicago. She is also the President-Elect of the American Society of Hematology. She has a broad background in clinical and translational research, with extensive experience in investigator initiated as well as multicenter sutdies primarily in the thalassemia and sickle cell disease. Dr. Thompson will examine advances in nonmalignant pediatric hematology focused on sickle cell and thalassemia in the context of her career perspective and opportunities for advocacy. 

Relevant financial relationships to disclose; discussion of off-label drug use: Amgen/Baxalta/Blue Bird Bio:Celegene/ Eli Lilly/Mast/Novartis: Research support, investigator; Novartis/ApoPharma/Celgene: Honorarium, consultant

(4003-04) Young Investigator Award Presentations

Adam L. Green, MD

(4003) Mechanism of Action and Combination Therapy Studies on the XP01 Inhibitor Selinexor in Pediatric High-Grade Glioma and Diffuse Intrinsic Pontine Glioma

Objective: Optimize the clinical use of selinexor in pediatric high-grade glioma and diffuse intrinsic pontine glioma through preclinical studies focused on mechansim of action and combination therapy. 

Chandrika Gowda, MD

(4004A) Regulation of C-KIT Oncogene by Ikaros and Casein Kinase II (CK2) in T Cell Acute Lymphoblastic Leukemia (T-ALL) and (4004B) Lim Domain Only Protein 2 (LMO2) Expression Is Regulated by karos and Casein Kinase II (CK2) in Acute Lymphoblastic Leukemia

Objectives:

It has been hypothesized that expression of c-kit is necessary for leukemia stem cell survival and c-kit inhibitors have been tested for the treatment of human leukemia. The mechanisms that regulate c-kit expression in T-ALL remain unknown. Here, we present evidence that expression c-kit in T-ALL is regulated at the transciptional level by Ikaros, a tumor suppressor whose deletion is associated with T-ALL development.

Overexpression of LM02 in T-ALL is associated with poor prognosis. The mechanisms that regulate LM02 expression in T-ALL are still unknown. Ikaros is a tumor suppressor protein that is encoded by IKZF1, whose deletion is associated with development of T-ALL. Casein Kinase II (CK2) oncogene impairs Ikaros function which can be restored by using CK2 inhibitors. The objective of this study is to investigate the role of Ikaros and CK2 in regulation of LM02 in leukemia. 

No relevant relationships and no dicussion of off-label drug use. 

No relevant financial relationships and no discussion of off-label drug use: Adam L. Green; Chandrika Gowda

Objectives:

1. Discuss pathways to successfully achieve career development goals.
2. Review the experiences of experts in the field.
3. Provide trainees and early career members an opportunity to network with leaders in the field of pediatric hematology/oncology.

Attendees will participate in discussion groups led by experts in defined areas of interest. The purpose of this luncheon is to provide early-career professionals a forum to discuss issues one-on-one with leaders in the field, ask questions related to career development in a small-group setting, and receive positive reinforcement regarding career goals specific to their interests. One to two discussion leaders will be placed at each luncheon table, with the option for participants to switch tables for further discussion on a wide range of topics.

Advanced registration is required.

The Advanced Practice Provider Special Interest Group is dedicated to providing a forum for the exchange of ideas and information among advanced practice providers (APPs) and for promotion of their role in the field of pediatric hematology and oncology. Our inaugural meeting in 2016 identified a strong need for enhanced educational opportunities for this population. At this year’s meeting, results of a formal needs assessment will be reviewed, and specific educational programming will be developed for the coming year.

Advanced registration is recommended.

Objectives:

1. Describe the accuracy and utility of currently available clinical scoring tools and plain radiographs in the early detection of arthropathic changes in asymptomatic or mildly symptomatic patients with hemophilia.
2. Provide an overview of the newest developments and applications of point-of-care musculoskeletal ultrasound in joint health surveillance and for early detection of joint disease in hemophilia.
3. Discuss the risks, benefits and limitations of joint MRI in the diagnosis, follow-up and management of hemophilic arthropathy in the pediatric population.

Arthropathy is the most common and serious chronic complication of hemophilia and early intervention is vital. Using an interactive review of actual cases, this workshop will address the existing challenges and future directions in the evaluation of hemophilic arthropathy. The panelists will review the current knowledge on clinical scoring tools, radiography, and MRI and provide the audience with recent data on the utility of point-of- care musculoskeletal ultrasound in the detection of early joint disease.

Objectives:

1. Summarize the fundamental principles of nuclease-mediated genome editing.
2. Recognize the potential clinical applications as well as the current limitations of nuclease-mediated genome editing.
3. Appraise the ethical implications of the use of therapeutic genome editing. Novel gene therapy strategies have demonstrated preclinical and early clinical efficacy.

Novel gene therapy strategies have demonstrated preclinical and early clinical efficacy. However, their further development has been hampered by low integration frequency and the possibility of inappropriate integration into deleterious sites. Nuclease-based genome editing platforms (e.g., ZFNs, TALENS, and CrispR/Cas9) enable more precise, higher-efficiency repair of targeted genes. Because many pediatric hematology/ oncology–related disorders could benefit from these approaches, pediatric hematology/oncology providers need to understand these technologies and their clinical applications, as well as recognize their limitations and ethical implications of integrating these techniques into clinical practice.

Objectives:

1. Define prognostic factors and the current risk stratification system for pediatric patients with liver tumors.
2. Describe the current treatment recommendations, including surgical guidelines, for pediatric patients with liver tumors.
3. Outline current international collaborations and resources for the treatment of pediatric liver tumors.

Pediatric livers tumors are rare and collaboration is necessary to best treat these tumors. Partnerships with surgeons optimize therapy and international collaborations have led to the identification of new prognostic factors and biological characteristics. Many oncologists are unaware of the nuances of current risk stratification and treatment procedures of these tumors. This session will update oncologists on the treatment, staging, and risk stratification of liver tumors and discuss a new online international consultation service.

Objectives:

1. Discuss new approaches related to diagnosis and treatment in pediatric hematology/oncology.
2. Examine current research outcomes from a variety of the latest in basic and clinical research.

Leukemia
Room 517D

Moderators: Christopher C. Porter, MD and Valerie I. Brown, MD PhD

Chimeric Antigen Receptor (CAR)-Modified T Cells (CTL019) Induce Durable CNS Remissions in Children with CNS/Combined Bone Marrow and CNS Relapsed/Refractory CD19+ Acute Lymphoblastic Leukemia (4005)
Mala K. Talekar, MD

Preclinical Efficacy of Daratumumab for T-ALL (4006)
Karen Bride, MD PhD

Minimal Residual Disease Assessment of Remission after Induction Therapy is Superior to Morphologic Assessment for Risk Stratification in Childhood Acute Lymphoblastic Leukemia: A Report from the Children’s Oncology Group (COG) (4007)
Sumit Gupta, MD PhD

Mertk is a Potential Therapeutic Target in Early Thymic Precursor Acute Lymphoblastic Leukemia (4008)
Ryan J. Summers, MD

No relevant relationships and no discussion of off-label drug use: Ryan J. Summers; Sumit Gupta

No relevant relationships, discussion of off-label drug use: Karen Bride; Mala K. Talekar

Solid Tumors/Lymphoma
Room 519

Moderators: Linda McAllister-Lucas, MD PhD and Nobuko Hijiya, MD

Risk Adapted Therapy Utilizing Upfront Brentuximab Vedotin (Bv) and Rituximab (R) with Reduced Toxicity Chemotherapy in the Treatment of Children, Adolescents and Young Adults (CAYA) with Newly Diagnosed Hodgkin Lymphoma (HL) (4009)
Jessica Hochberg, MD

Excellent Outcomes following Response-based Omission of Radiotherapy in children and Adolescents with Intermediate or Advanced Stage Hodgkin Lymphoma (4010)
Nmazuo Ozuah, MBBS

Targeting Integrin-mediated Signaling in Metastatic Ewing Sarcoma (4011)
Jade Wulff, MD

The Transcriptional Co-activator TAZ is a Potent Mediator of Alveolar Rhabdomyosarcoma Tumorigenesis (4012)
Michael Deel, MD

No relevant relationships and no discussion of off-label drug use: Jessica Hochberg; Nmazuo Ozuah; Jade Wulff; Michael Deel

Hematology I
Room 524

Moderators: Lindsay Mize, MSN RN CPNP and Emily R. Meier, MD

Decreased Microvascular Perfusion is a Physiological Biomarker of Vaso-occlusive Crisis due to Mental Stress and Pain Anticipation in Sickle Cell Disease (4013)
Saranya Veluswamy, MD

Realizing Effectiveness across Continents with Hydroxyurea (REACH): Enrollment and Baseline Characteristics (4014)
Patrick McGann, MD MS

Reduced Glutamate Channel Phosphorylation in Pain-related Brain areas in a Transgenic Model of Sickle Cell Disease (4015)
Dana M. LeBlanc, MD

Quick Start Hydroxyurea Initiation project (Q-SHIP): Targeted Education after Crisis to Increase the use of Hydroxyurea in Children with Sickle Cell Anemia (4016)
Lydia H. Pecker, MD

No relevant relationships and no discussion of off-label drug use: Saranya Veluswamy; Dana M. LeBlanc; Lydia H. Pecker

No relevant relationships, discussion of off-label drug use: Patrick McGann

Objectives:

1. Describe the mechanisms of action, predictive biomarkers and adverse events of T cell checkpoint inhibitors and their rationale therapeutic combinations.
2. Discuss how myeloid cells affect cancer growth and cancer therapy, and how they might be therapeutically targeted.
3. Identify strategies to enhance the function of anticancer antibodies and CAR T cells.

The emerging successes of antibodies and cellular therapies highlight the power of the immune system. The immunologic landscape of cancer also involves numerous innate and adaptive immune mechanisms such as tumor-associated macrophages, NK cells, and T-cell checkpoints that are emerging as bone fide cancer biomarkers and therapeutic targets. We will discuss and review pediatric perspectives on these emerging mechanisms’ roles, giving the participants practical knowledge for studying and deploying the next generation of cancer immunotherapies.

Objectives:

1. Discuss pathways to successfully achieve career development goals.
2. Review the experiences of experts in the field.
3. Provide trainees and early career members an opportunity to network with leaders in the field of pediatric hematology/oncology.

Venous thromboembolism (VTE) increasingly is being recognized in children, often as a complication of complex medical conditions, such as congenital heart disease, nephrotic syndrome, inflammatory bowel disease, sickle cell anemia, etc. Up front management, the duration of therapy, and the role of thromboprophylaxis in these high-risk cohorts may be nuanced. This interactive case-based session will equip participants with up-to-date, evidence-based knowledge on the epidemiology, clinical presentation, and management of VTE in selected high- risk populations, using a focused mini-review approach.

Objectives:

1. Increased awareness of how cultural and racial differences affect health outcomes.
2. Improved understanding of how our biases affect patient trust and outcomes and develop tools for improvement.
3. Develop strategies to increase diversity within our field.

Racial healthcare inequity is most certainly a multifactorial problem. Barriers to healthcare equity include the healthcare system (insurance, funding, whiteness, social determinants), the patient (poor health literacy, fear, mistrust), the community (awareness, advocacy), and healthcare providers (unconscious bias, attitudes, racism, stereotyping). We need to increase our awareness of these issues and how they negatively impact our patients of color. We, as medical care providers, potentially have the greatest power to address our biases to help close the gap for our underrepresented and marginalized families.

Children’s Oncology Group
Room 517D

Moderator: Patrick A. Brown, MD

Outcomes of Children, Adolescents, and Young Adults with Acute Lymphoblastic Leukemia based on Blast Genetics at Diagnosis: A Report from the Children’s Oncology Group (4017)

Mignon Loh, MD

Effects of Sodium Thiosulfate on Prevention of Cisplatin-Induced Hearing Loss in Children with Cancer: Results of the Randomized Controlled Trial ACCL0431 (4018)
David R. Freyer, DO MS 

COG Neuroblastoma Clinical Trials: Recent Data and Future Plans (4019)
Julie Park, MD

Stem Cell Transplantation—Advancing the Field Through Collaboration (4020)
Michael Neider, MD

No relevant relationships and no discussion of off-label drug use: Mignon Loh; David R. Freyer

No relevant relationships, discussion of off-label drug use: Julie Park

Relevant relationships; discussion of off-label drug use: Michael Neider

Outcomes Research
Room 524

Moderators: Karen E. Effinger, MD MS, and Rochelle Maxwell, MD

Ruxolitinib in Pediatric Patients with Severe of Refractory Graft-Versus-Host Disease: A Single-Institution Case Series (4021)
Julie R. Boiko, MD MS

Patient and Parent Experience of Cancer Symptoms as Assessed by the Memorial Symptom Assessment Scale for Children (4022)
Hayden Leeds, BA

Impact of Ethnicity and Education Level on Parental Preferences for Information Giving and Decision Making in the Pediatric Cancer Setting (4023)
Katie Bergstrom, MS CGC

Processing Speed and Academic Fluency in Youth with Sickle Cell Disease (4024)
Molly Gardner, PhD

No relevant relationships and no discussion of off-label drug use: Hayden Leeds; Katie Bergstrom; Molly Gardner

No relevant relationships, discussion of off-label drug use: Julie R. Boiko

Objectives:

1. Introduce the concept, the challenge of defining, and the increasing scope of social media.
2. Provide an overview of types of social media currently utilized by providers and educators in the healthcare delivery network.
3. Engage attendees in discussions regarding the pros and pitfalls of social media in healthcare delivery and discuss legal and ethical considerations including ownership of content, patient and provider privacy, effects on personal and patient engagement/communication, etc.

Understanding the benefits of social media can be an invaluable asset to a post-doctoral clinical fellow becoming a junior faculty member or to a senior level educator in engaging a younger audience. However, it also is important to be cognizant of the legal/ethical considerations when discussing/using social media in the context of the patient-provider relationship, the transfer of information between providers, and how it may blend into external work-personal relationships. This session will enable honest dialogue using small-group discussions and a series of case vignettes. The moderators will emphasize strengths of and obstacles to overcome when engaging with social media.

No relevant financial relationships to disclose and no discussion of off-label drug use: Mona D. Shah; Vandy Black; Dominder Kaur

Objectives:

1. Improved understanding of management techniques for priapism.
2. Develop strategies for management of chronic pain in SCD associated with vasocclusive crisis and AVN.
3. Describe contraceptive options for the AYA population with SCD and best practices for hydroxyurea use.

Survival of children with sickle cell disease (SCD) has improved over the past 30 to 40 years thanks to early identification of affected infants through newborn screening, early institution of penicillin prophylaxis, and family education. Despite the increased utilization of hydroxyurea in childhood, end organ damage continues to occur, resulting in morbidity and decreased quality of life as patients age. Pediatric hematologists often lack experience caring for chronic problems that more commonly are associated with adult patients, despite taking care of the adolescent and young adult (AYA) population. This session will address three common AYA SCD-related issues.

Objectives:

1. Discuss the approach to a pediatric patient with a rare sarcoma.
2. Review the latest molecular diagnostic tools for accurate diagnosis of soft tissue sarcoma.
3. Review the role of chemotherapy and targeted therapy in the management of three molecularly defined pediatric sarcomas.

As a result of rapid advances in molecular diagnostic techniques, soft tissue sarcomas increasingly have been classified on the basis of underlying molecular alterations. This workshop will focus on three molecularly defined pediatric sarcomas and update the audience on latest advances in diagnosis and management of these tumors. We will present examples of some early successes in targeted therapy. After this session, attendees will have a better understanding of the principals of managing these tumors.

Objectives:

1. Identify mechanism(s) of T-cell activation and inhibition and discuss new therapeutic approaches of checkpoint blockade in pediatric cancers.
2. Increase our understanding of mechanism(s) TGFB-1 inhibition by the innate immune response in Neuroblastoma and new methods of circumvention.
3. Describe the mechanisms of resistance to immunotherapy in childhood ALL, and to suggest strategies to overcome resistance for therapeutic benefit.

The tumor microenvironment recently has been identified to regulate the immune response to a variety of malignancies. A number of mechanisms including T-cell checkpoint blockade, TGFB-1 inhibition, and receptor downregulation have been identified as key components of suppressing immune responses to a number of malignancies. Pediatric oncologists and mentees in training have limited information regarding the mechanisms, pathophysiology, and methods of circumvention of immunosuppression by the tumor microenvironment.

Objectives:

1. Describe the review process for a submitted manuscript.
2. Identify what reviewers and editors consider as they review manuscripts.
3. Recognize which types of manuscripts are most appropriate for which journals.

Although scientific publication is a critical component of academic medicine and is essential for career advancement, there is little formal instruction on how the publication process actually works. Peter E. Newburger, MD, and Thomas Gross, MD PhD, the editors of Pediatric Blood & Cancer, the premier journal in our field, will provide their insights into the editorial and review process to help trainees and junior faculty enhance the likelihood of successfully getting published.

Objectives:

1. Discuss the utility of next generation sequencing for FVIII and FIX genes.
2. Describe the characteristic of individuals with GATA2 mutations.
3. Summarize the role that PIKC3A mutations play in the development of lymphatic malformations.

Advances in technologies to DNA sequencing have aided the discovery of the genetic basis for many inherited hematologic diseases. In addition, detailed genotype/ phenotype mapping of known genes is feasible for clinical outcomes. Pediatric hematologists/oncologists may not have the most recent information regarding genes that cause inherited myelodysplastic syndrome or vascular malformations. In addition, they may not have current knowledge of the genotype/phenotype information that is being studied in hemophilia A and B.

New genetic information is accumulating at a rapid pace. How genetic information about well-characterized conditions such as hemophilia may impact clinical phenotypes will be of value to practitioners. For patients with bone marrow failure, providers’ knowledge of the genetic basis may impact prognosis and treatment approach. The genetics of vascular malformations is a rapidly advancing field and an understanding of mutations in signaling pathways can lead to pharmacologic strategies for treatment.

Objectives:

1. Recognize the scope of changes occurring at a national level in our sub-specialty.
2. Identify challenges around matching number of trainees with number of jobs open.
3. Envision potential new training, employment and staffing models within the PHO field.

Pediatric hematologists/oncologists and ASPHO see that the pediatric hematology/oncology workforce is changing. In 2015, based on data collected by the ASPHO Practice Committee and the Workforce Task Force, ASPHO formed the Workforce Strategy Advisory Group to synthesize data and share this information with the field. In this session, speakers will summarize the findings of that group.

No relevant financial relationships to disclose and no discussion of off-label drug use: Jeffrey Hord; Patrick Leavey; Adman L. Green; Rachel Thienprayoon; Michelle Hermiston; Elizabeth Villavicencio

Relevant financial relationships to disclose; discussion of off-label drug use: Ayesha Zia- Shire/Octapharma: Advisory Board Member, honoraria

Objectives:

1. Delineate the risk factors associated with opportunistic viral and fungal system infections in the immunocompromised pediatric cancer patients.
2. Identify the safety and efficacy of anti-fungal antibiotics in immunosuppressed pediatric cancer patients.
3. Increase our understanding of new antiviral antibiotics in the treatment of respiratory viral diseases in immunocompromised pediatric cancer patients.

Pediatric cancer patients are at high risk for developing opportunistic viral and fungal systemic infections. Recent advances have been made in identifying the risk factors as well as new antiviral and antifungal antibodies and genetically re- engineered immune cells to treat these high-risk opportunistic viral and fungal systemic infections. Pediatric hematologists/ oncologists and mentees in training have limited information on the risk factors of and uses for new antimicrobial drugs and genetically re-engineered immune cells to treat these infections.

Objectives:

1. Describe the mutations and pathophysiology of LCH.
2. Describe the targeted therapies available, their efficacies and limitations.
3. Identify individualized treatment algorithms for patients with refractory/recurrent disease.

Recent discoveries have revolutionized our understanding of the pathogenesis of LCH and related histiocytic diseases and opened up avenues for targeted therapies. However, we have limited data on the safety and efficacy of these newer targeted therapies in children with LCH. The decision on when and how to utilize which agent is a complex one. The goals of this workshop are to review (1) the results of mutational studies and the pathophysiology of LCH, (2) available targeted therapies and the preliminary efficacy results, and (3) treatment algorithms for refractory/recurrent disease, including second- and third-line chemotherapy agents and targeted therapies.

Physician burnout is a complex issue within the pediatric hematology/oncology workforce that can affect patient care, professionalism, physician care and safety, and the healthcare system. A growing body of evidence shows successes using multiple strategies, both personal and organizational, to build physician resilience to prevent and/or decrease burnout. This session will incorporate audience self-assessment, a didactic presentation, and a panel discussion to provide attendees with the tools needed to prevent and decrease burnout, promote personal resilience, and maintain a healthy environment for healthcare providers and their patients.

No Relevant financial relationships to disclose and no discussion of off-label drug use: Amy L. Billett; John D. Mahan; Carolline A. Hastings, MD; Akiko Shimamura, MD PhD; Eric S. Sandler, MD; Glen Lew, MD

Objectives:

1. Assess genomic testing approaches to identify appropriate therapies.
2. Apply population-based genome epidemiology to individualize patient care.
3. Interpret and manage secondary findings revealed through genomic testing.

“Precision medicine” is a broad term which, in pediatric oncology, can be applied to everything from cancer diagnosis to treatment. As our understanding of the genomics of cancer development and treatment progresses, the challenge for the clinical provider is how to translate new data to patient care. This workshop provides an overview of this challenge and three perspectives on how to use the latest discoveries to make pediatric oncology a more precise practice.

1. Educate membership on advocacy practice, including commonly used language and the legislative process
2. Describe ASPHO’s current federal policy advocacy activities and potential impact on pediatric hematology/oncology practice.
3. Introduce ASPHO membership to advocacy skills with opportunity for direct application with policymakers.

ASPHO’s strategic plan states that ASPHO will be recognized by the global pediatric hematology/oncology community as advocating for pediatric hematology/oncology. The Board of Trustees has invested resources into revamping the Advocacy Committee, developing ASPHO’s advocacy priorities, and hiring staff to move ASPHO’s advocacy agenda forward. Attendees will understand the impact of policies affecting patients and the profession and how to engage in the advocacy process.

No relevant financial relationships to disclose and no discussion of off-label drug use: Jordan Wildermuth; Natasha Frederick; Naomi Luban; Robert S. Nickel

Objectives:

1. Discuss factors unique to immunocompromised pediatric hematology/oncology patients that should be taken into account when ECMO is considered.
2. Discuss the role of noninvasive ventilation in this population in the context of the available literature and expert opinion.
3. Increased knowledge of the use of renal replacement strategies in critically ill immunocompromised pediatric hematology/oncology patients.

Immunocompromised hematology or oncology patients often require critical care intervention because of their underlying disease or complications of therapy. The outcome for these patients has improved in recent years, but remains unacceptably low. In this workshop, we will discuss and debate changes in the critical care management of hematology, oncology, and hematopoietic cell transplant patients that may have led to improved survival. Topics will include the timing of critical care intervention, use of ECMO, non-invasive ventilation, and fluid management strategies.

No relevant financial relationships to disclose and no discussion of off-label drug use: Georges Rivard

How I Treat...a Palliative Oncology Subspecialty Panel Common issues related to the integration of palliative care into pediatric oncology will be addressed by pioneers and experts in the field. The mission of the Palliative Care SIG is to accelerate the availability of high-quality palliative care to pediatric oncology patients across the country. Advanced registration is recommended.

The MOC learning session will feature an American Board of Pediatrics (ABP) Lifelong Learning and Self-Assessment Module. It is intended not only for those who are enrolled in the MOC process and would like to earn MOC activity points, but also for anyone wishing for a broad review of recent literature relevant to pediatric hematology/oncology. The session will be conducted in an interactive group setting led by authors or experts in their fields. The session will cover multiple-choice questions in the 2017 Pediatric Hematology-Oncology Self-Assessment. Please bring your laptop to enter the answers to each question during the session. At the completion of this session, those enrolled in the ABP MOC program will submit their answers to the online module for scoring to receive 10 MOC Part 2 points and 3 AMA PRA Category 1 Credits™. Enrolled participants can access the module(s) online from the ABP website at www.abp. org. For additional information about ABP’s MOC program requirements, visit www.abp.org or call the ABP Contact Center at 919.929.0461. Please note that this is not a Board review activity; the workshop is designed to facilitate completion of ABP’s Lifelong Learning and Self-Assessment MOC requirement. Advanced registration is required.

The registration fee is $50.

No relevant financial relationships to disclose; discussion of off-label drug use: Zora Rogers; Sheila Weitzman

No relevant financial relationships to disclose and no discussion of off-label drug use: Glen Lew; ZoAnn Dreyer; Karen Effinger; Emily R. Meier; Michael A. Pulsipher; Elizabeth Raetz; Naomi Winick