A virtual roundtable discussion with noted investigators Prof Claire Harrison and Dr John Mascarenhas for a review of recent innovations in the treatment of myeloproliferative neoplasms.

Novel and Emerging Therapeutic Strategies in the Management of Hematologic Disorder-Related Anemia — Part 1: Our interview with Dr Komrokji highlights the following topics and cases from his practice.

• Approved and emerging therapies for patients with transfusion-dependent myelodysplastic syndromes (MDS); mechanism of action of the investigational erythroid maturation agent (EMA) luspatercept (00:00)
• Design, entry criteria and outcomes of the Phase III MEDALIST trial of luspatercept for the treatment of anemia in patients with very low-, low- or intermediate-risk MDS with ring sideroblasts who require red blood cell (RBC) transfusions (2:10)
• Clinical experience with the EMAs luspatercept and sotatercept (6:03)
• Potential FDA approval of luspatercept for the management of low-risk MDS (9:11)
• Ongoing investigation of luspatercept-based strategies for MDS (12:25)
• Investigation of venetoclax in combination with a hypomethylating agent for higher-risk MDS (16:00)
• Role of luspatercept in myelofibrosis; predictors of benefit from luspatercept (18:24)
• Case: A man in his mid-70s presents with fatigue and dyspnea and is diagnosed with lower-risk MDS with ring sideroblasts (21:17)
• Initial workup and diagnosis for patients with MDS (23:58)
• Risk stratification in MDS and therapeutic options for patients at lower versus higher risk (25:52)
• Clinical experience with EMAs for lower-risk, RBC transfusion-dependent MDS (29:42)
• Monitoring and management of iron overload in patients with lower-risk, RBC transfusion-dependent MDS (32:30)
• Case: A man in his early 60s with lenalidomide-refractory, lower-risk MDS and a del(5q) mutation receives the telomerase inhibitor imetelstat on a clinical trial (36:09)
• Case: A man in his mid-70s with postpolycythemia vera myelofibrosis with anemia and splenomegaly receives initial ruxolitinib therapy (40:49)
• Activity and tolerability of JAK1/2 inhibitors in myelofibrosis (45:09)
• Case: A woman in her mid-70s with myelofibrosis and a JAK2 mutation presents with progressive splenomegaly and anemia and receives ruxolitinib (48:31)
• Novel agents and strategies under investigation for myeloproliferative neoplasms (MPNs) (50:40)
• Incidence of IDH1/2 mutations in patients with MPNs; responses to IDH1/2 inhibitors and ongoing clinical investigations (53:13)
• Activity of the antifibrotic immunomodulator PRM-151 in patients with myelofibrosis (56:04)
• Common misconceptions about the use of erythropoietin-stimulating agents (57:45)
• Perspective on the appropriate choice of therapy for patients with intermediate-2 or high-risk myelofibrosis (1:01:41)
• Lack of correlation between JAK2 mutation status and response to ruxolitinib (1:03:19)

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Novel and Emerging Therapeutic Strategies in the Management of Hematologic Disorder-Related Anemia — Part 1: Our most recent one-on-one interview with Dr Komrokji featuring emerging research and cases from his practice.

Novel and Emerging Therapeutic Strategies in the Management of Hematologic Disorder-Related Anemia — Part 2: Our interview with Dr Cappellini highlights the following topics and cases from her practice.

• Pathophysiology of thalassemia (00:00)
• Evolution of therapies for thalassemia (4:20)
• Prevalence and incidence of thalassemia (9:23)
• Classification and genetic inheritance of thalassemia (11:52)
• Novel treatments under investigation for thalassemia; role of gene therapy and EMAs (19:27)
• Emerging data with luspatercept and sotatercept for patients with thalassemia (23:15)
• Results of the Phase III BELIEVE trial evaluating luspatercept versus placebo for adult patients with beta thalassemia who require regular RBC transfusions (26:28)
• Tolerability and side effects associated with luspatercept (28:35)
• Ongoing Phase II BEYOND study evaluating the efficacy and safety of luspatercept in adults with beta thalassemia who are not transfusion dependent (32:37)
• Activity of luspatercept in patients with anemia of chronic disease and MDS (33:48)
• Effect of luspatercept on quality of life for patients with thalassemia (35:43)
• Optimal selection of patients with thalassemia who would benefit from luspatercept (39:11)
• Gene therapy for transfusion-dependent beta thalassemia (42:06)
• Gene therapy process for patients with thalassemia (45:26)
• Perspective on novel approaches to the treatment of thalassemia (51:16)

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Novel and Emerging Therapeutic Strategies in the Management of Hematologic Disorder-Related Anemia — Part 2: Our most recent one-on-one interview with Dr Cappellini featuring emerging research and cases from her practice.

Proceedings from a CME symposium held at the 2019 ASCO Annual Meeting. Featuring perspectives from Prof Georgina Long and Drs Jason J Luke and Jeffrey S Weber.

• Introduction (00:00)
• • Program overview: Dr Love
• Adjuvant Systemic Therapy of Melanoma (1:33)
• Case (Dr Weber): A man in his early 50s with melanoma and a BRAF V600E tumor mutation who received adjuvant dabrafenib/trametinib (7:20)
• Case (Prof Long): A man in his mid-60s with BRAF wild-type melanoma who developed a local recurrence after adjuvant nivolumab (9:28)
• Case (Dr Luke): A woman in her mid-40s with BRAF wild-type melanoma who received adjuvant nivolumab and developed immune-related hypothyroidism (15:22)
• BRAF/MEK Inhibitor Combinations in the Management of Metastatic Melanoma (24:52)
• Case (Prof Long): A woman in her mid-70s with newly diagnosed metastatic melanoma and a BRAF V600K tumor mutation who received encorafenib/binimetinib (37:40)
• Case (Dr Luke): A man in his mid-50s with metastatic melanoma and a BRAF tumor mutation who developed brain metastases (42:02)
• Immune Checkpoint Inhibitors in the Management of Metastatic Melanoma (48:25)
• Case (Dr Weber): A man in his late 30s with metastatic BRAF wild-type melanoma, including brain metastases, who experienced disease progression on single-agent anti-PD-1 antibody therapy and was switched to nivolumab/ipilimumab (50:22)

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Proceedings from a CME symposium held at the 2019 ASCO Annual Meeting. Featuring perspectives from Prof Georgina Long and Drs Jason J Luke and Jeffrey S Weber.

A presentation from Dr O’Shaughnessy featuring emerging research and cases from her practice.

• Introduction (00:00)
• • Program overview: Dr Love
• Estrogen and Progesterone Receptors; Clinical Use of Endocrine Treatment (1:20)
• Case: A woman in her early 60s with ER/PR-positive, HER2-negative breast cancer (BC) who experienced intolerable arthralgias with adjuvant aromatase inhibition and was switched to tamoxifen (7:08)
• First-Line Endocrine Therapy for Metastatic Disease: Role of CDK4/6 Inhibitors (12:44)
• Case: A woman in her mid-60s with ER/PR-positive de novo metastatic BC (mBC) who received palbociclib/letrozole (12:54)
• Case: A woman in her mid-50s with ER/PR-positive, HER2-negative BC who developed metastatic disease after receiving 4 years of adjuvant letrozole and started treatment with ribociclib/fulvestrant (27:39)
• Case: A woman in her early 60s who received abemaciclib/exemestane for ER/PR-positive, HER2-negative mBC to the brain and bone (33:09)
• Second-Line Endocrine Therapy for Metastatic Disease: Role of mTOR Inhibitors (42:18)
• Case: A woman in her late 60s with ER/PR-positive, HER2-negative mBC who received exemestane/everolimus after disease progression on a CDK4/6 inhibitor (42:26)
• New Approaches Under Investigation (51:47)
• Assessing and Optimizing Treatment Adherence (59:00)
• Case: A woman in her early 30s with ER/PR-positive, HER2-negative BC who demonstrated poor adherence to adjuvant endocrine therapy because of weight gain (59:09)

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A presentation from Dr O’Shaughnessy featuring emerging research and cases from her practice.

Proceedings from a CME symposium held at the 2019 ASCO Annual Meeting. Featuring perspectives from Prof Dirk Arnold and Drs Tanios Bekaii-Saab, Joseph Chao, Anthony El-Khoueiry, Richard S Finn, Yelena Y Janjigian, Scott Kopetz and Eileen M O’Reilly.

• Introduction (00:00)
• Program overview: Dr Love
• Hepatocellular Carcinoma (HCC) — Dr El-Khoueiry and Dr Finn (2:01)
• Case (Dr Gujja): A man in his mid-70s with Child-Pugh A multifocal HCC and a tumor thrombus in the midhepatic vein extending into the floor of the right atrium (2:12)
• Case (Dr Park): A woman in her early 70s with Child-Pugh B HCC who developed hand-foot syndrome and required a dose reduction while receiving first-line sorafenib and now has progressive disease (16:11)
• Pancreatic Adenocarcinoma (PAD) — Dr Bekaii-Saab and Dr O’Reilly (41:01)
• Case (Dr Ibrahim): A man in his late 50s with symptomatic metastatic PAD who experienced a dramatic response to modified FOLFIRINOX (52:14)
• Cases (Dr Park and Dr Morganstein): Elderly patients with metastatic PAD, poor performance status and disease progression on nab paclitaxel/gemcitabine (1:00:22)
• Case (Dr Gujja): A woman in her late 60s with metastatic PAD and a germline BRCA2 mutation (1:06:00)
• Gastric/Gastroesophageal (GEJ) Cancers — Dr Chao and Dr Janjigian (1:20:30)
• Case (Dr Favaro): A man in his late 30s with HER2-positive metastatic gastric cancer who received paclitaxel/ramucirumab after disease progression and worsening neuropathy on FOLFOX/trastuzumab (1:35:52)
• Case (Dr Morganstein): A man in his mid-60s with microsatellite-stable, PD-L1-negative metastatic GEJ cancer who attained a complete response to second-line nivolumab (1:39:34)
• Colorectal Cancer (CRC) — Prof Arnold and Dr Kopetz (1:52:56)
• Case (Dr Morganstein): A woman in her early 40s with widespread RAS wild-type, microsatellite-stable, left-sided metastatic CRC (mCRC) (1:57:43)
• Case (Dr Ibrahim): A woman in her late 70s with MSI-high mCRC who refused chemotherapy and experienced a completed response to pembrolizumab (2:04:01)
• Case (Dr Favaro): A woman in her late 80s with RAS wild-type, microsatellite-stable, right-sided mCRC with a BRAF V600E tumor mutation who received BRAF-targeted therapy (2:13:49)

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