ASPHO Webinars Connect Learners to the Latest in Pediatric Hematology/Oncology Clinical Treatment, Best Practices, and Professional Development

Speakers:

• Natalie Bezler, MD (moderator)
• Erin McGuire Pastor, DO
• Anna Vinitsky, MD

Although there is no standard education or training for Quality improvement (QI) work it remains both a fellowship graduation requirement as well as yearly expectation for PHO providers to engage in such projects for maintenance of certification (MOC 4). Many PHO providers lack education in the fundamental basics of QI research, nor have they had exposure to the appropriate tools for such work to be successful. Using case-based learning we aim to discuss QI projects that were born from medical errors, near misses and gaps in healthcare for PHO patients. We hope these examples easily be transitioned to other institutions as the chosen cases are generalizable from hospital to hospital. We will discuss how we have used these projects for MOC part 4 credit as well as how that incentive has led to increased provider buy in.

Learning Objectives:

1. Use case-based review to describe process behind developing and implementing QI projects in pediatric hematology oncology.
2. Identify opportunities for using your institutional QI work to obtain part 4 credits for MOC.

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Speakers

• Taizo Nakano, MD (moderator)
• Joana Mack, MD
• Bhuvana Setty, MD

Sirolimus has emerged as a promising therapeutic option for patients with complex vascular anomalies, offering hope in conditions previously considered untreatable. However, while this mTOR inhibitor shows significant efficacy, its use comes with unique challenges, including potential side effects and the need for long-term management. With evolving research and clinical experience, it's crucial for clinicians to stay informed on how best to balance the benefits and risks of Sirolimus therapy.

This webinar will provide an in-depth exploration of the latest evidence on Sirolimus use in vascular anomalies, from its mechanisms of action to practical considerations for patient management. We will review the most recent data on efficacy, dosing protocols, and safety profiles, as well as strategies to mitigate complications like immunosuppression and delayed wound healing. Case studies will be used to illustrate successful treatment approaches and common pitfalls. We will review lessons learned from the historical use of sirolimus in the field of Pediatric Hematology/Oncology.

Clinicians will also gain insights into how to assess the appropriateness of Sirolimus for different vascular anomalies, personalize treatment plans, and navigate ongoing care. Special focus will be placed on discussing quality-of-life considerations, including patient counseling, the potential need for lifelong therapy, and managing patient and family expectations. Join us for a comprehensive guide on optimizing outcomes with Sirolimus therapy in the evolving field of vascular anomalies.

The objectives for this webinar are below:

Learning Objectives:

1. Understand the Therapeutic Benefits and Mechanism of Action of Sirolimus in Treating Vascular Anomalies - Learners will be able to describe how Sirolimus works to manage vascular anomalies and the clinical improvements observed in patients undergoing this treatment.
2. Identify and Manage the Common and Severe Complications Associated with Sirolimus Therapy - Learners will recognize potential complications and adverse effects of Sirolimus, such as immunosuppression, wound healing issues, and elevated cholesterol, and discuss strategies for their management.
3. Evaluate the Risk-Benefit Profile of Sirolimus in Different Types of Vascular Anomalies - Learners will analyze case studies or clinical trial data to assess when Sirolimus therapy is most beneficial and when its risks may outweigh its potential benefits for patients with varying presentations of vascular anomalies.

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Speakers:

• Neethu M Menon, MD
  • UT Health Science Center at Houston
• Suchitra Acharya, MD
  • Hemostasis and Thrombosis Center, Northwell Health
• Oluyemisi Adeyemi-Fowode, MD
  • Texas Pediatric and Adolescent Gynecology, Houston

Description:

According to NHANES data reviewed by Weyand et al (Prevalence of IDA and ID in US females aged 12-21y, 2003-2020. JAMA Network 2023), prevalence of iron deficiency anemia (IDA) among adolescents with potential to menstruate is high at 6.3%, with the prevalence of latent iron deficiency (ID) even higher at 17% using a ferritin cut-off of < 15ng/ml and 38.6% using a cut-off of <25ng/ml. The largest contributor to this, heavy menstrual bleeding (HMB), is not a topic addressed routinely during fellowship, despite the fact that it is caused by an inherited bleeding disorder in 20% of cases (HMB in Adolescents: ACOG Management Recommendations. Am Fam Physician 2020). Major gaps globally in the treatment of HMB are attributed at least partly due to limited physician awareness (Reflections on access to care for heavy menstrual bleeding: Past, present, and in times of the COVID-19 pandemic. Int J Gynaecol Obstet 2023). There is increasing collaboration between hematologists, adolescent medicine specialists and gynecologists across the country to manage this problem through multidisciplinary clinics, but education on this topic is largely missing at the trainee level and hence an important knowledge gap that will be addressed in this webinar.

The objectives for this webinar are below:

Learning Objectives:

1. Review the epidemiology of iron deficiency (ID) and related anemia (IDA) in adolescents with potential to menstruate.
2. Highlight updates in the diagnosis and management of ID/IDA, including parenteral iron therapy and hormonal therapy for heavy menstrual bleeding.
3. Discuss complications associated with IDA and heavy menstrual bleeding like missed school/ work, interference with activities of daily living, depression, and exacerbation of other adolescent health issues.

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Description:

Increased incorporation of germline genetic testing in pediatric oncology is uncovering cancer predispositions in more and more children. It is thus imperative that pediatric hematology oncology providers know which children warrant testing and what to do with the resulting information. Towards this end, it is debated whether children should be tested for germline variants in adult onset cancer predisposition genes. And for those who do test positive, does the information gained provide insights into tumor formation and/or provide new strategies for therapeutic intervention? As relates to cancer surveillance, it is often challenging to know when and how to monitor children with underlying predispositions and whether such monitoring is truly effective.

Further, are there more sensitive and accurate ways to screen for cancers in individuals at risk? In light of the increased availability and use of germline genetic testing, it is pediatric hematology oncology providers must know when and how to appropriately counsel, test and manage patients with underlying predispositions. This webinar will discuss new advances in the field to update current knowledge and spur conversations about these exciting topics.

Learning Objectives:

1. Understand when and if testing for cancer predisposition should be offered to children and how identified variants influence cancer risk

2. Recognize the benefits and challenges of surveillance across the full spectrum of cancer predisposition syndromes

3. Know how testing for cell-free DNA can enhance cancer detection in individuals with Li Fraumeni syndrome

Speakers:

Kim Nichols, MD (moderator)
Christian Kratz, MD
Melissa Perrino, MD
David Malkin, MD

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Description:

Bone marrow transplant (BMT) has over the decades offered a curative option for sickle cell disease (SCD), a multisystem disorder that leads to end-organ damage, decreased quality of life and survival. However, less than 20% of patients have a suitable HLA matched sibling donor and concerns about toxicity have limited its use. The recent FDA approval of two gene therapy (GT) products, which offer transformative effects on disease amelioration and quality of life, have added to the armamentarium of treatment options for patients. The availability of these therapies has not necessarily led to equitable access for patients. Several factors limit access to these therapies including the cost of these therapies, geographic accessibility of GT/BMT centers, concerns about toxicity and lack of data about the long-term durability of the newer gene therapy products. Healthcare providers and patients/families are challenged in decision-making about which curative/transformative therapy to select given the lack of data showing comparative effectiveness of the newer therapies compared to BMT. This webinar will address these challenges in the US while offering suggestions to ensure equitable access for patients living with SCD.

Pediatric hematologists serve as trusted sources of information for treatment options for patients with sickle cell disease and are gatekeepers for access to these therapies. Thus, it is important for pediatric hematologists to be knowledgeable about these therapies including the challenges faced by patients/families as they consider these therapies, and to have insights into their own biases, to facilitate decision-making about these therapies that is patient/family centered and offers equitable access to these therapies.

Learning Objectives:

1. Describe the current landscape in the US for access to BMT, gene therapy and editing techniques for SCD.
2. Describe patient and healthcare provider perspectives about gene therapy for SCD.
3. Describe the challenges related to achieving equitable access these therapies and suggestions for improving access.

Speakers

• Maa-Ohui Quarmyne, MBChB MS (moderator) - Phoenix Children's Hospital
• Lakshmanan Krishnamurti, MD - Yale School of Medicine
• Nitya Bakshi, MBBS MS - Yale School of Medicine
• Elizabeth Stenger, MD – Children’s Healthcare of Atlanta

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