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Medscape
7 hours ago
- Health
- Medscape
S2 Episode 5: Posttransplant Monitoring in Myelofibrosis
This transcript has been edited for clarity. For more episodes, download the Medscape app or subscribe to the podcast on Apple Podcasts, Spotify, or your preferred podcast provider. Tania Jain, MBBS: Hello everyone. We're back in our myelofibrosis for Medscape InDiscussion podcast. This is season two, and this is episode five, where we'll talk about transplant outcomes in myelofibrosis and how to address relapse post-transplant. I'm Tania Jain, and today we have our very special guest, Dr Rachel Salit, who is an associate professor at Fred Hutchinson Cancer Center and the University of Washington School of Medicine. As a hematologist-oncologist, Dr Salit specializes in stem cell transplant, focusing on improving outcomes for patients with myeloproliferative neoplasms. Her research has included developing clinical trials to enhance and improve transplant success, prevent graft-vs-host disease (GVHD), and she also works on initiatives to support patients' return-to-work journeys post-transplant. Welcome, Dr Salit. It's an honor to have you. Rachel Salit, MD: Thank you for having me today. Jain: What we want to talk about today is pre-transplant, post-transplant, and how to think about transplant. I think we would all agree that transplant is presented and thought about differently, to some extent, by different clinicians and others who see patients in the clinic. What is your approach, and how do you go about telling patients about the role of transplant? Salit: The way I approach patients with myelofibrosis is from two different sides. One is the scoring system criteria — going over the Dynamic International Prognostic Scoring System (DIPSS) and the Mutation-Enhanced International Prognostic Scoring System (MIPSS-70). That would include what their blood counts look like, their molecular profile, cytogenetics, whether they have any peripheral blasts, and whether we think they're at imminent danger for transforming to leukemia or needing blood or platelet transfusions. The second side I look at with them is their life goals — what they are hoping for in terms of longevity vs quality of life. I think we've made great strides in transplant in the last 10 years in myelofibrosis since the approval of Janus kinase (JAK) inhibitors. I talk with them about the role of JAK inhibitors in the pretransplant setting — whether they've had one or not — depending on their symptoms and splenomegaly, and how that affects transplant timing. Ultimately, it's their choice. Patients often have strong opinions. There are patients with high-risk disease by scoring systems, but who are having a rather good quality of life and don't want to rush into treatment. Then there are patients with lower risk but who, because of younger age or life goals, want to proceed more expediently. I let the patient guide me. The third component that goes into it is what our donor search looks like. I often don't know that when I first meet the patient, so I always add the caveat: This is the conversation we're having now, but once we assess sibling matches or unrelated donors, risks and benefits might change. Jain: One of the things that challenges me is that there's a lot of prediction involved in the transplant decision, especially the first part you mentioned, about leukemia progression risk. There's no perfect metric to predict someone's transplant outcomes, whether for disease control, preventing relapse, or even nonrelapse mortality. How do you play the prediction game, without a crystal ball? Salit: I think it is one of the most challenging aspects of myelofibrosis. I empathize with patients in that we consider this a somewhat elective procedure that no one wants to need. It's tougher on them than it is for acute myeloid leukemia (AML) patients, where we say, 'This is your option, and you need to do it now.' For me, I try to look at the disease kinetics. When did it develop? How long has it been stable? If they have peripheral blasts in their blood, have they stayed at 1% for 2 years, or have they risen to 2% or 3%? Have their counts stayed stable? Is their spleen growing? Are their symptoms increasing? If patients have poor-risk factors but stable disease, I'm less aggressive than I am with patients who have more benign features but rapidly progressing disease. Sometimes in younger patients, the disease progresses faster than expected, especially if they have just one high-risk mutation. Jain: I always say that one of the most important things to follow is the trajectory of disease. When counts begin to drop or blasts start to appear, that makes you pause and think about transplant, or at least do a bone marrow biopsy to help make that decision. I want to shift gears. I know you've been involved in defining remission post-transplant, which is a huge unmet need. Many disease features don't resolve quickly after transplant, yet we consider the transplant successful. The forms and shapes that remission or recurrence can take post-transplant have evolved. Can you share how you got involved in unifying the concept of remission post-transplant and what insights you can offer for our audience? Salit: I got involved in this from two angles. Clinically, it's long been unsatisfying to tell patients post-transplant that they have low counts, persistent fibrosis, or splenomegaly when we discharge them from the acute transplant service at day 100. The providers we discharge them to — often general oncologists — aren't always sure what to say. Are you in remission? How long will it last? How do we test for it? The second part came from my work on the Medicare–Center for International Blood and Marrow Transplant Research (CIBMTR)) composite study, looking at whether transplant benefits patients over 55. We've been working on this for the past 7 years. When we met to define the response to transplant, we used the newly developed International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria for drug response in myelofibrosis, and we found that to be very complex for CIBMTR reporting. The people reviewing charts were not reporting consistently. I was approached by Wael Saber from CIBMTR to help develop a consistent remission definition so that centers reporting outcomes — remission, persistent disease, or relapse — could be compared. We want to draw conclusions about what regimens and donors are working best. We recognized that, unlike AML patients who go into transplant in CR1 (first complete remission), myelofibrosis patients go into transplant with active disease: persistent blasts, fibrosis, splenomegaly. We formed a committee of myelofibrosis transplant experts from the US and Europe. We wanted both CIBMTR and the European Society for Blood and Marrow Transplantation (EBMT) to have the same criteria so we could collaborate and combine data. The strongest takeaway was that the main thing transplant can do that drugs currently cannot is put patients into molecular remission. Most patients have driver mutations — JAK2, CALR, MPL — and we know the majority clear these mutations post-transplant. We recognized that patients with molecular remission may still have low counts or morphologic abnormalities. We classified these as subsets of molecular remission. We also agreed that persistent molecular abnormalities — especially if decreasing — shouldn't be considered relapse. Persistent disease can clear over time with rising CD3 chimerism and immune suppression taper. We reserved 'relapse' for patients who had previously cleared their mutation and then redeveloped it. That's a much more concerning scenario requiring intervention. Jain: That makes a lot of sense. One way I explain it to patients is that a transplant is an intervention with curative potential, but disease elimination takes time, even after donor hematopoiesis is established. It's not an immediate clinical remission — spleen and counts take time to improve. But once donor hematopoiesis is present, that can eventually trigger graft-vs-leukemia activity to eliminate residual disease. Do you know if there's a difference in how CIBMTR and EBMT capture relapse? Salit: I honestly don't know. But one of the goals of our project, with about six stakeholders from each registry, was to create a uniform definition. I think the recent New England Journal of Medicine paper by Gagelmann and colleagues spearheaded this molecular remission definition. CIBMTR and Dr Saber are very enthusiastic about this, and I think we can develop reporting forms that consistently capture remission, persistent disease, and relapse as three categories. We acknowledge there will be missing data. Not every center regularly tests molecular driver mutations. We've also discussed the role of chimerism. Fractionated chimerism is important, but most European centers don't assess it. They can do weekly molecular tests; we might test every 3 months at best. So, blood counts and chimerism will still play a role. I've piloted this approach with my patients, and they find it more satisfying. At day 100, I can say, 'You still have fibrosis, splenomegaly, and low platelets — but your JAK2 is negative. We're considering this remission.' That's much more reassuring for them than wondering why we even did the transplant. Jain: I totally agree. Bringing together the American and European efforts — making them globally unified — would be a huge advancement in research. Right now, it's hard to use or compare registry data across systems since it's not apples to apples. You mentioned you're already using this with your patients. There are times when spleen, fibrosis, or counts aren't fully normalized. Can you share your approach for relapse or 'impending relapse' when you see persistent disease features like cytopenias, transfusion needs, splenomegaly, dropping chimerism, or persistent molecular mutations? Salit: We check molecular driver mutations every 3 months — at months 3, 6, 9, and 12. If the 3-month result is negative but the patient still has low CD3 chimerism, needs transfusions, or has fibrosis, we start tapering immune suppression at day 100. We follow chimerism monthly during tapering. If it's not at 100% by 6 months, we may do a bone marrow biopsy to see if anything concerning is present. If the mutation's variable allele frequency is decreasing, that's reassuring. If it's back and chimerism is dropping — say it was 100% at day 100 and 90% at 6 months — we consider donor lymphocyte infusion (DLI), assuming they're off immune suppression and don't have GVHD. If they're still on immune suppression, we taper conservatively and wait a month before giving DLI. We only use therapy like hypomethylating agents and JAK inhibitors if blasts return in the peripheral blood or marrow, or if abnormal cytogenetics reappear. Jain: Those are tough situations — when blasts are visible, or disease features reappear. How often do you consider a second transplant in myelofibrosis? Salit: Not often. Thankfully, we're seeing only 5%-10% relapse rates. At our center, we're still doing relatively mild ablative transplants: Cytoxan/busulfan for those under 60, or decitabine/melphalan for those over 60. If a patient completely loses CD34 or CD33 chimerism, we consider a second transplant. There's concern that giving DLI in that setting could cause aplasia. If the disease burden is too high, we go straight to the second transplant. Jain: That makes sense. Well, this was phenomenal. Thinking about transplant is a nuanced process, and we loved hearing your thoughts. We look forward to reading about the remission definitions post-transplant and implementing them in the clinic. On behalf of our audience, thank you so much for your time. That concludes episode 5 on transplant outcomes in myelofibrosis. We'll see you in the next episode. Listen to additional seasons of this podcast. Primary Myelofibrosis Role of Hematopoietic Stem Cell Transplantation in Patients With Myeloproliferative Disease Decreasing Chronic Graft-Versus-Host Disease Rates in All Populations Diagnosis and Evaluation of Prognosis of Myelofibrosis: A British Society for Haematology Guideline Prognostic Value of Blasts in Peripheral Blood in Myelofibrosis in the Ruxolitinib Era Acute Myeloid Leukemia (AML) CIBMTR Myelofibrosis Medicare Study Proposals for Revised International Working Group-European LeukemiaNet Criteria for Anemia Response in Myelofibrosis Utility of Assessing CD3+ Cell Chimerism Within the First Months After Allogeneic Hematopoietic Stem-Cell Transplantation for Acute Myeloid Leukemia Clearance of Driver Mutations After Transplantation for Myelofibrosis Donor Lymphocyte Infusion and Molecular Monitoring for Relapsed Myelofibrosis After Hematopoietic Cell Transplantation Medscape © 2025 WebMD, LLC Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape. Cite this: Posttransplant Monitoring in Myelofibrosis - Medscape - Jun 10, 2025.
Globe and Mail
16-05-2025
- Health
- Globe and Mail
Aplastic Anemia Market to Witness Significant Growth by 2034, Fueled by Rising Prevalence and a Strong Pipeline
The aplastic anemia treatment market is poised for significant growth, with key pharmaceutical companies including Novartis, Pfizer, Teva Pharmaceuticals, Kyowa Kirin, Regeneron Pharmaceuticals, Gamida Cell, and others leading therapeutic innovation in this rare hematologic disorder. DelveInsight's " Aplastic Anemia Market Insight, Epidemiology And Market Forecast - 2034" report provides an in-depth understanding of aplastic anemia, including historical and forecasted epidemiology as well as market trends across the US, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. According to the comprehensive analysis, the aplastic anemia market, valued at approximately USD 270 million in 2023 across the 7MM, is projected to expand substantially through 2034, fueled by increasing disease incidence, growing uptake of existing therapies, and the anticipated launch of promising pipeline candidates. Furthermore, the US currently dominates the aplastic anemia treatment landscape, accounting for approximately 70% of the market share in the 7MM, followed by Japan with the second-highest market value at approximately USD 35 million in 2023. Among European nations, Germany leads the market, with additional growth anticipated across all regions during the forecast period. Download the Aplastic Anemia Market Forecast Report to understand which factors are driving the Aplastic Anemia market @ Aplastic Anemia Market Trends. DelveInsight's aplastic anemia epidemiological analysis reveals that Japan reported the highest aplastic anemia incident cases, contributing approximately 30% of cases across the 7MM in 2023. Notably, 56% of aplastic anemia cases were reported in individuals aged 60 and above, while 43% were below the age of 60. Discover evolving trends in the Aplastic Anemia patient pool forecasts @ Aplastic Anemia Epidemiological Analysis. The current aplastic anemia treatment varies based on patient age and disease severity. Hematopoietic stem cell transplantation (HSCT) remains the standard of care for patients under 40 years, while immunosuppressive therapy (IST) is preferred for those above 40 years. Novartis' PROMACTA / REVOLADE (eltrombopag) currently leads market revenue, followed by various immunosuppressive therapies. However, younger patients experience significantly better long-term survival compared to those aged 60 and above, highlighting a critical unmet need for improved management strategies in older populations. The aplastic anemia therapeutic landscape is evolving with several marketed and emerging options. ALVAIZ (eltrombopag choline) from Teva Pharmaceuticals (NYSE: TEVA), an oral thrombopoietin receptor agonist that stimulates platelet production, approved in November 2023, represents an important treatment advancement. In Japan, Kyowa Kirin's ROMIPLATE has gained approval for patients showing inadequate response to conventional therapy. The patent for PROMACTA (eltrombopag), a key treatment for aplastic anemia, is set to expire on July 13, 2025. This expiry will likely lead to the introduction of generic versions, which can reduce treatment costs and increase patient access. As Promacta is currently a leading therapy in the aplastic anemia market, the availability of generics is expected to intensify competition, lower prices, and potentially shift prescribing patterns. Looking toward future market growth, promising pipeline candidates include REGN7257 from Regeneron Pharmaceuticals (NASDAQ: REGN), a gamma chain cytokine receptor antibody targeting the common gamma chain found in various interleukin receptors. Currently in Phase I/II clinical trials, this therapy aims to address T-cell-mediated pathogenesis in severe aplastic anemia patients who have relapsed after immunosuppressive therapy. Another noteworthy emerging therapy is OMISIRGE (omidubicel) by Gamida Cell (NASDAQ: GMDA), a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood. In April 2025, CK0801, an allogeneic T regulatory (Treg) cell therapy manufactured by Cellenkos, received FDA Orphan Drug Designation for aplastic anemia, supporting its development as a novel treatment for this rare condition. A Phase III registration trial is planned for the second half of 2025, positioning CK0801 as a promising non-immunosuppressive option for aplastic anemia patients unresponsive to standard therapies. Discover evolving trends in the Aplastic Anemia treatment landscape @ Aplastic Anemia Recent Developments. Despite therapeutic advances, significant challenges remain in the aplastic anemia treatment landscape. Finding effective and well-tolerated treatments for patients who do not respond to standard therapies or who are unsuitable candidates for aggressive interventions continues to be problematic. Additionally, poor survival rates among older patients represent a substantial unmet medical need requiring innovative approaches. Table of Contents 1. Key Insights 2. Executive Summary of Aplastic Anemia 3. Aplastic Anemia Competitive Intelligence 4. Aplastic Anemia: Market Overview at a Glance 5. Aplastic Anemia: Disease Background and Overview 6. Patient Journey 7. Aplastic Anemia Epidemiology and Patient Population 8. Treatment Algorithm, Current Treatment, and Medical Practices 9. Aplastic Anemia Unmet Needs 10. Key Endpoints of Aplastic Anemia Treatment 11. Aplastic Anemia Marketed Products 12. Aplastic Anemia Emerging Therapies 13. Aplastic Anemia: Seven Major Market Analysis 14. Attribute analysis 15. 7MM: Market Outlook 16. Access and Reimbursement Overview of Aplastic Anemia 17. KOL Views 18. Aplastic Anemia Market Drivers 19. Aplastic Anemia Market Barriers 20. Appendix 21. DelveInsight Capabilities 22. Disclaimer 23. About DelveInsight Related Reports Aplastic Anemia Pipeline Insight Aplastic Anemia Pipeline Insight provides comprehensive insights about the Aplastic Anemia pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the Aplastic Anemia companies, including Gamida-Cell, Regeneron Pharmaceuticals, and BioLineRx, among others. About DelveInsight DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Arpit Anand Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: Nevada Country: United States Website:
Daily Mail
10-05-2025
- Health
- Daily Mail
Top doctor reveals five sneaky things people have no idea cause cancer
A leading oncologist has revealed the five common habits that could increase your risk of developing cancer. Dr Mikkael Sekeres, chief of hematology at the Sylvester Comprehensive Cancer Center in Florida, recently warned unexpected day-to-day activities may lead to cancer developing in the body. While an explosion of colon cancers in people under 50 has made headlines in recent years, government scientists have now found 14 other types of cancers are also rising among the young. In a recent comprehensive review of more than 2million young cancer patients diagnosed between 2010 to 2019, researchers found 14 cancers were on the rise: Stomach, colon, pancreatic, bone and joint, melanoma, female breast, cervical, uterine, testicular, kidney and four types of cancers of the lymph system. While it's well known cigarette smoking and alcohol can cause cancer, even seemingly healthy drinks like hot tea could cause damage and lead to throat cancer. And though one in three Americans have tattoos, some studies suggest getting one could raise the risk of lymphoma. Even dyeing your hair has been linked to hormone-sensitive cancers like breast and ovarian cancer. Writing for the Washington Post, Dr Sekeres cautioned 'none of these factors has evidence on par with risks like drinking alcohol,' but they may warrant further research to determine exactly how risky they are. Below, reveals the five common activities that may raise the risk of cancer: Excessively hot drinks It's well known that alcohol can increase the risk of several forms of cancer, as it breaks down into acetaldehyde, a toxic chemical that damages DNA and creates inflammation. However, even drinking hot beverages like tea may raise the risk of throat cancer. A study from China found people who drank burning-hot tea - over 140 degrees Fahrenheit (60 degrees Celsius) - and at least one alcoholic beverage every day had a five-fold greater risk of esophageal cancer than those who did not. And drinking the hot tea alone every day doubled the risk. Additionally, research from the UK found almost twice the risk of throat cancer in people who drank four to six cups of hot coffee or tea every day compared to those who didn't drink it at all. Drinks above 140 degrees Fahrenheit are thought to damage cells in the esophagus when the tea is consumed multiple times a day for years. Dr Sekeres said: 'It's reasonable to avoid regular intake of any very hot beverage, though it is more important for you to stop smoking and avoid drinking alcohol.' Grilling As summer weather sets in, millions of Americans are firing up their grills. However, Dr Sekeres warns against eating meats cooked at high temperatures, such as hot dogs or burgers. He warns grilling meats like beef, poultry, fish or pork at high temperatures generates heterocyclic amines and polycyclic aromatic hydrocarbons - groups of chemical compounds that are known to be mutagenic and potentially carcinogenic. Dr Sekeres wrote: 'This occurs as the meat proteins react to the heat and as fat and juice drip onto the fire's surface, causing flames and smoke.' Recent research suggests chemicals created by these meats trigger harmful inflammation and oxidative stress, an imbalance between disease-fighting antioxidants and harmful free radicals, which attack cells and tissues. This increases the risk of colon cancer, which is surging in young Americans. The Department of Health and Human Services has determined some polycyclic aromatic hydrocarbons may 'reasonably be expected to be carcinogens' and some people who have breathed or touched mixtures of these chemicals for long periods of time have developed cancer. In animal studies, animals breathing in air or ingesting food containing polycyclic aromatic hydrocarbons developed lung cancer and stomach cancer. Meanwhile, heterocyclic amines may contribute to cancer by causing gene mutations and causing the new cells to grow in an uncontrollable manner to form a tumor. In animal studies, rats that ingested heterocyclic amines developed cancers of the mammary glands, colon and pancreas. Dr Sekeres limits his high-heat cooked meat to once a month, even during grilling season. He wrote: Because there is strong evidence that red and processed meat is linked to cancer, my advice would be to limit grilling and consuming those in particular. 'Less grilling is better, and each of us must determine our own tolerance for cancer risk when deciding how often to consumed grilled meats.' Chemical hair straighteners and dyes Dyeing or straightening your hair could also increase your risk of developing cancer. Dr Sekeres explains this is because hair dye and chemical straighteners contain formaldehyde, a proven carcinogen in humans, and phthalates, toxic chemicals used to make plastics more flexible and durable. They are also shown to be endocrine-disrupting chemicals, meaning they imitate the body's hormones and interfere with the production of - and response to - natural hormones like estrogen and testosterone. This increases the risk of developing hormone-sensitive cancers like breast and ovarian cancer. One 2020 study of sisters found those who used permanent hair dye were more likely to develop breast cancer than those who never used it. Another 2022 study found using any straightening products within the previous year led to a higher chance of developing uterine cancer. Dr Sekeres wrote: Though the data are limited, I suggest reviewing the contents of your chemical hair products before using them, to see if they contain formaldehyde or endocrine-disrupting chemicals.' Tattoos One in three Americans has at least one tattoo, and 22 percent have more than one, according to recent data from Pew Research Center. While the procedure is generally considered harmless, a recent study suggested tattoos could increase the risk of lymphoma - a type of cancer in the lymphatic system, a network of vessels and glands that are part of the immune system. Researchers in Sweden found tattooed people had a 21 percent higher risk of the cancer than people without ink. The team suggested potentially carcinogenic chemicals in the ink activate an immune response when injected. The exact list of chemicals depends on the ink brand, but there are usually synthetic molecules, stabilizers and heavy metals like cadmium, which has been linked to kidney damage, bone disease, lung disease and an increased risk of certain cancers. Additionally, a small Danish study of twins found those with tattoos were 62 percent more likely to develop skin cancer, as chemicals in the ink may accumulate in the lymph nodes, glands that are a vital part of the body's immune system. The dye could trigger chronic inflammation, leading to abnormal cell growth and an increased risk of cancer, the researchers said. However, Dr Sekeres cautioned both studies are small and can't prove direct causation. He said: 'Even if the link is true, because lymphoma is a rare cancer diagnosis, affecting just 21 per 100,000 people in the United States yearly, the chances of developing lymphoma from getting a tattoo would be quite small.' Hookah smoking Shisha or hookah smoking involves burning flavored tobacco with charcoal and then breathing in the tobacco smoke using a mouth pipe. Recent research suggests one in 100 Americans have smoked hookah at least once. While many believe this form of smoking is healthier than smoking cigarettes because they think the water in the device filters the smoke, studies have shown this is a common misconception. Hookah smoke has high levels of harmful chemicals including tar, carbon monoxide, heavy metals and cancer-causing chemicals called carcinogens. Dr Sekeres said hookah smokers are exposed to more carbon monoxide than people who smoke cigarettes because sessions last longer, around one to two hours, compared to a few minutes for cigarettes. In fact, the FDA estimates during a one-hour long hookah session, users inhale up to 200 times more smoke than with a single cigarette. Dr Sekeres cites a 2024 study of 40,000 people from northern Vietnam that found hookah smokers had elevated risks of dying from liver, lung, head and neck, and stomach cancers over an average of 11 years compared to those who didn't smoke. Dr Sekeres wrote: 'Scientists are confident that tobacco intake is linked to an increased cancer risk. My recommendation: Avoid all forms of tobacco, including smoking cigarettes and hookah.'
