Rare and Orphan Diseases

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Anthony Stein, MD

Could a novel triplet combination therapy offer a new path forward in acute myeloid leukemia (AML) treatment? A recent Phase 1B trial explored the potential of tagraxofusp, azacitidine, and venetoclax for AML patients. To discuss the findings on this triplet combination therapy, Dr. Charles Turck speaks with Dr. Anthony Stein, Professor in the Department of Hematology and Hematopoietic Cell Transplantation at City of Hope in Duarte, California.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Yi Bin Chen, MD

Guest: Olaf Penack, MD

For the past several decades, standard treatment for acute graft-versus-host disease (aGVHD) has been systemic high-dose steroids.1 While essential in some ways to initially control aGVHD, steroids are not beneficial in the long run due to the immunosuppression and toxicities associated with high cumulative doses.1,2 So what can be done moving forward to improve first-line steroid response and minimize cumulative steroid exposure? Joining Dr Charles Turck to discuss the potential of nonimmunosuppressive steroid-sparing agents for the first-line treatment of aGVHD are Drs Yi Bin Chen and Olaf Penack. Dr Chen is the Director of the Hematopoietic Cell Transplant and Cell Therapy Program at Massachusetts General Hospital, and Dr Penack is a senior physician and principal investigator within Hematology and Oncology at Charité Berlin.

References:

1. Bell EJ, Yu J, Bhatt V, et al. Healthcare resource utilization and costs of steroid-associated complications in patients with graft-versus-host disease. Transplant and Cell Ther. 2022;28(10):707.e1-707.e7.
2. Martin PJ, Rizzo JD, Wingard JR, et al. First- and second-line systemic treatment of acute graft-versus-host disease: recommendations of the American Society of Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2012;18(8):1150-1163.

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Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Jason Ackrivo, MD, MSCE

Respiratory complications are fairly common in patients with neuromuscular disorders, and given their vast impacts on a patient’s prognosis and quality of life, it’s critical to proactively detect and monitor any signs of respiratory decline. To learn more about the symptoms, impacts, and diagnosis of respiratory complications in patients with neuromuscular disorders, Dr. Charles Turck speaks with Dr. Jason Ackrivo. Not only is Dr. Ackrivo the Associate Director of the Fishman Program for Home Assisted Ventilation, but he's also an Assistant Professor of Medicine in Pulmonary, Allergy, and Critical Care at the Hospital of the University of Pennsylvania.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Bimaje Akpa, MD

Clinical practice guidelines from the American College of Chest Physicians provide recommendations for managing respiratory complications in patients with neuromuscular diseases, which can help inform our evaluation and treatment decisions. Joining Dr. Charles Turck to break down key insights from the CHEST guidelines and their impacts on clinical practice is Dr. Bimaje Akpa. Dr. Akpa is an Assistant Professor of Medicine in the Division of Pulmonary, Allergy, Critical Care, and Sleep Medicine at the University of Minnesota.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Hank Mayer, MD

Patients with neuromuscular conditions often exhibit respiratory symptoms that impact their airway clearance and gas exchange, making it increasingly difficult for them to breathe. However, effective assessment techniques and early intervention can help mitigate the progression of this dysfunction, potentially keeping patients from needing extended recovery time or even inpatient care. Joining Dr. Charles Turck to discuss the importance of informed, proactive intervention for respiratory symptoms in patients with neuromuscular conditions is Dr. Hank Mayer. Dr. Mayer is the Medical Director of the Pulmonary Function Laboratory at the Children's Hospital of Philadelphia and a Professor of Clinical Pediatrics at the University of Pennsylvania's Perelman School of Medicine.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Miguel-Angel Perales, MD

Guest: Anna Sureda, MD, PhD

Unfortunately, the treatment paradigm for acute graft-versus-host disease (aGVHD) has not changed for a couple of decades as steroids remain the first-line treatment.1 However, steroids are not a targeted therapy, and they have many side effects that are not well tolerated by patients, including life-threatening infections.1-3 Given these risks, it is important to know how to manage infections related to immunosuppression and optimize the care of patients with aGVHD. Joining Dr Charles Turck to discuss the current landscape of aGVHD management are Dr Miguel-Angel Perales and Dr Anna Sureda. Dr Miguel-Angel Perales is the Chief of the Adult Bone Marrow Transplantation Service at Memorial Sloan Kettering Cancer Center in New York, and Dr Sureda is the Head of Clinical Hematology at the University of Barcelona in Spain.

References:

1. Bell EJ, Yu J, Bhatt V, et al. Healthcare resource utilization and costs of steroid-associated complications in patients with graft-versus-host disease. Transplant Cell Ther. 2022;28(10):707.e1-707.e7. doi:10.1016/j.jtct.2022.04.014
2. Sullivan PW, Ghushchyan VH, Globe G, Sucher B. Health-related quality of life associated with systemic steroids. Qual Life Res. 2017;26(4):1037-1058.
3. García-Cadenas I, Rivera I, Martino R, et al. Patterns of infection and infection-related mortality in patients with steroid-refractory acute graft versus host disease. Bone Marrow …

Guest: Gil I. Wolfe MD, FAAN

Rozanolixizumab is a fast-acting myasthenia gravis treatment that reduces IgG levels by blocking the neonatal Fc receptor. Dr. Gil Wolfe explains how it compares to other treatment options like corticosteroids, taking side effects and quality of life into consideration. Dr. Wolf is a SUNY Distinguished Professor of Neurology at the University of Buffalo Jacobs School of Medicine and Biomedical Sciences.

Guest: Sudipto Mukherjee, MD, PhD, MPH

This series of bite-sized episodes will take you on the winding journey that patients with idiopathic multicentric Castleman disease (iMCD) often undergo to achieve an accurate diagnosis. Once the condition is finally identified, even the most astute specialists can find themselves challenged by the treatment, management, and monitoring of these patients. Join Drs. Corey Casper and Sudipto Mukherjee as they explain the ups and downs of this rare disease.

Guest: Mateo Bustamante

Idiopathic multicentric Castleman disease (iMCD) is a rare, life-threatening condition. Early diagnosis through excisional biopsies is key for speeding up treatment and improve patient outcomes. Learn more with Mateo Bustamante, a Senior Clinical Data Analyst at the Center for Cytokine Storm Treatment and Laboratory at University of Pennsylvania’s Perelman School of Medicine.

Guest: Joshua Brandstadter, MD, PhD, MSc

Castleman disease is difficult to diagnose and treat, with half of patients not responding to therapy. To address these challenges, Dr. Joshua Brandstadter discusses his research on how stromal cells play a role in the disease. Based at the University of Pennsylvania, Dr. Brandstadter is an Instructor of Medicine in the Division of Hematology and Oncology and the Director of Clinical Research for the Center of Cytokine Storm Treatment and Laboratory.