In this patient advocate episode, Ms. Barbara Pruitt shares what it's like to live with immune thrombocytopenia (ITP), a condition she has had since age four. Barbara takes us through more than six decades of treatments, from childhood steroids and a splenectomy to today's newer therapies. She describes growing up without meeting anyone else with ITP and the moment a patient organization changed that. She also talks about the everyday adjustments that let her keep traveling, dancing, and enjoying life, and how a hematologist's words can either wound or empower. Hosts Drs. Ronak Mistry and Vivek Patel explore what clinicians can learn from her story, including why quality of life matters as much as lab values. Barbara closes with advice for anyone newly diagnosed.

Key Takeaways:

1. Treat the diagnosis as an emotional event, not just a clinical one. Barbara reminds physicians to acknowledge the emotional weight of the diagnosis, and connect patients to peer communities and support resources early, especially children and teens.
2. Look beyond the platelet count. Barbara's current therapy hasn't raised her count, but less bruising and fewer petechiae make it worth continuing. She urges clinicians not to abandon a therapy over a small fluctuation and encourages listeners to ask about other symptoms of ITP including fatigue, bleeding, and energy, which are often dismissed. Ask about symptoms and function at every visit, and weigh them alongside the number when judging whether a treatment is working.
3. Plan the treatment around the patient's life.Consider how treatment options impact our patient’s quality of life. Present all reasonable options with their pros and cons, and think through practical fit, such as injections versus oral drugs and food interactions. Invite questions about activities and alternative therapies, because patients will otherwise go looking on their own.

For additional ASH resources related to ITP, please consider visiting our ASH Clinical Practice Guidelines on Immune Thrombocytopenia web page.

This episode was supported by Sanofi.

In this episode of HemeTalks, we are joined by Ms. Elise Burton, who shares her experience living with paroxysmal nocturnal hemoglobinuria (PNH). Elise describes how her journey began several years earlier with a diagnosis of aplastic anemia, before her condition was later recognized as PNH after she developed evidence of unexplained hemolysis. She walks through her treatment journey, including eculizumab, ravulizumab, and enrollment in a danicopan clinical trial, as well as the side effects she experienced, the financial navigation required to stay on therapy, and the routine health maintenance that can be displaced when managing a rare disease becomes all-consuming. She also discusses the patient communities and advocacy organizations that supported her. Our patient advocate episodes highlight the importance of advocacy, community, and shared understanding between patients and their health care providers.

Key Takeaways:

1. Preemptive anticipatory counseling is critical for patients, particularly those with rare diseases, to ensure rapid access to life-saving care in emergencies. Elise shares how her hematologist made sure she understood her condition and gave her essential contact information, along with a letter detailing her condition to share with healthcare professionals if she ever needed emergency medical attention. Equipping patients with a plan before a crisis can meaningfully change the care they receive.

2. Rare disease care can become a part-time job, and general health maintenance can get lost. Patients with chronic hematologic diseases have medical needs beyond their hematologic condition. Hematologists can help by acknowledging the administrative burden, connecting patients with organizations such as AAMDS, NORD, The Assistance Fund, and the Patient Advocate Foundation, and reinforcing the importance of routine health screenings.

To access the PNH patient resources mentioned in this episode, please refer to the link provided here.

This episode was supported by Alexion.

Dr. Cece Calhoun (Yale School of Medicine) joins Heme Talks to discuss one of the most vulnerable moments in sickle cell care: the transition from pediatric to adult management. Using a real case of a 24-year-old with hemoglobin SC disease re-establishing care after a lapse, Dr. Calhoun walks through how to build trust with adolescent and young adult patients, why sickle cell disease is a systemic vascular and inflammatory condition rather than "just pain," and how to explain complex pathophysiology in patient-friendly language. The conversation covers essential screening guidelines for new adult patients, strategies for community providers managing sickle cell without specialist access, the evolving role of hydroxyurea (including the PIVOT trial in hemoglobin SC disease), and a practical, patient-centered approach to outpatient pain management.

Clinical Pearls:

1. The first visit is about trust, not just data. Understanding a patient's life context, not just their labs is foundational, especially for AYA patients re-entering care after a gap; overwhelming a new patient with testing can undermine that trust.
2. Sickle cell disease is systemic, not just a localized vaso-occlusive crisis. Even "milder" genotypes like hemoglobin SC cause damage through chronic inflammation, hemolysis, and vascular interaction. The historical framing of SC as mild disease is outdated, and end-organ damage can appear early in young adults.
3. Structured screening and collaboration close care gaps. New adult patients need a full baseline workup (CBC/retic, electrophoresis, renal and liver labs, brain MRI, ophthalmology referral), and providers without sickle cell specialization should identify a partner institution and use published NHLBI/ASH guidelines rather than navigating complex cases alone.
4. Hydroxyurea remains foundational, even without perfect evidence in SC disease. It's the only FDA-approved therapy shown to prolong survival in sickle cell disease; while the PIVOT trial's data in hemoglobin SC disease is still limited, empiric use is reasonable in symptomatic patients showing signs of pain or end-organ damage.

For additional ASH resources related to Sickle Cell Disease, please consider visiting our ASH Clinical Practice Guidelines on Sickle Cell Disease web page.

This podcast episode is supported by Sanofi.