Frequently Asked Questions About Waldenström Macroglobulinemia

Editor’s Note: This guide was originally published by The International Waldenstrom’s Macroglobulinemia Foundation (IWMF) here.

By Peter DeNardis, Marcia Klepac, Elena Malunis, and Linda Nelson, 2017. Revised Linda Nelson, 2019.


Frequently_Asked_Questions_English_FINAL_COVERS-022021-1

What Health Disparities Exist for Patients with Renal Medullary Carcinoma?

This video was originally published by Diverse Health Hub here.

What Health Disparities Exist for Patients with Renal Medullary Carcinoma? from Diverse Health Hub on Vimeo.

Dr. Pavlos Msaouel broaches the disparities that exist for patients with Renal Medullary Carcinoma (RMC). He contends that RMC afflicts a very vulnerable population, that is young African Americans. The disparity exists due to the young age of these patients who aren’t part of a work force with health insurance. Secondarily, this has caused RMC to be underdiagnosed, preventing optimal treatment for patients.

On a global scale, Nigerians face the same disparity, as traits of RMC affect a large number of the population. There are virtually zero reports of its existence and patients again are underdiagnosed. Dr. Msaouel argues we need to research questions that answer where and how RMC affects particular populations in order to equalize the disparity of underdiagnosed patients. Currently the US is gathering data and creating RMC patient communities and advocates. Dr. Msaouel stresses this research is needed on a global scale as well.


Transcript:

Rebecca Law:

Dr. Msaouel, what disparities exist for patients with Renal Medullary Carcinoma?

Dr. Pavlos Msaouel:

Now that is a wonderful and very important question because there are certainly disparities in caring for individuals with RMC (Renal Medullary Carcinoma). Think about it these are people who are young and young people in general — you know they do not have necessarily for example, in the U.S. that kind of insurance when they’re in their twenties or teenagers that they will have later in life or even you know are not part of the work force in that sense. But it’s even more challenging if you think that the vast majority of individuals with RMC are young African Americans. So, this is a disease that particularly afflicts a very vulnerable population, so there is no doubt that health disparities afflict individuals with RMC and this may be part of the reason why for many years this was an underdiagnosed disease.

Dr. Pavlos Msaouel:

We are finding out now that this cancer is more common than we originally thought. It’s still a rare cancer, there is no doubt about that, but it is more common than we originally thought to the point that nowadays in our clinics we see about 1 new case, 1 new patient with RMC per month. So that’s certainly more common than the about 100 cases that have been published thus far in the literature—there are many more that we do not know of. And think about it also in a different way, from a more global perspective. So, there is about let’s say 3 million individuals in the U.S. who carry the sickle cell trait. Most would be African Americans, other ones will be Caucasians so there are many different people who can have the sickle cell trait, but it’s mainly African Americans, but there are 300 million people in the world that carry the sickle cell trait — mainly in Africa.

Dr. Pavlos Msaouel: 

So, let’s take for example, Nigeria – there are many people in Nigeria who have the sickle cell trait yet how may reports are there about the existence of this cancer in these countries, essentially almost 0 and so that is a disparity in itself. This is a cancer that can be difficult to diagnose especially if you do not know about it, if you’ve never heard of it, but even if you do – even if a physician or healthcare personnel knows about this cancer it still needs specific tests to be done and many of them cannot be done in most countries so that’s also a disparity.

Dr. Pavlos Msaouel:

So the mere fact that this cancer cannot be diagnosed in many countries in the world is a disparity in itself, so if we were able to correct this and understand more about where it happens and how often it happens we know much more nowadays thankfully through the work of many people in the U.S. now that are becoming passionate about helping individuals with RMC (Renal Medullary Carcinoma) so we are gathering a lot of data, a lot of information, we have patient communities in the U.S., we have patient advocates that help immensely in the U.S, but this is very likely a global phenomenon, so that’s a disparity that will need to be addressed.

How Can Providers Combat Inequities in the Care of Renal Medullary Carcinoma Patients?

This video was originally published by Diverse Health Hub here.

How Can Providers Combat Inequities in the Care of Renal Medullary Carcinoma Patients? from Diverse Health Hub on Vimeo.

Dr. Msaouel tackles the question of how providers can combat inequities in the care of Renal Medullary Carcinoma (RMC) patients. He states that patients go undiagnosed because providers don’t know about the disease. Dr. Msaouel urges that healthcare providers raise awareness about RMC and in doing so be diligent about screening patients with particular symptoms.

According to Dr. Msaouel, young African Americans possessing sickle cell traits and blood in the urine must be taken seriously and screened with ultrasound to check for suspicious kidney masses. They must be thoroughly checked with proper diagnosis. Dr. Msaouel says providers in the US know more about RMC but efforts must be increased to raise optimal awareness not only here in the states but around the world.


Transcript:

Rebecca Law: Dr. Msaouel, what can providers do to combat inequities in the care of Renal Medullary Carcinoma patients?

Dr. Pavlos Msaouel: So that’s a wonderful question. One way to be able to address this is to help as a provider raise awareness. So, one of the things that is happening is that very often in these cases, patients with RMC are undiagnosed because people don’t know about it. So, if healthcare providers know about this then they can think about it, and then they can start having it in mind. And so, if a young individual — especially if it’s a young African American who has the sickle cell trait — comes with blood in the urine in the ER you have to take it seriously.

Dr. Pavlos Msaouel: You have to do potentially an ultrasound of the kidney and if you do an ultrasound of the kidneys and you find something that’s suspicious like a mass, you have to take that very seriously. This is thankfully happening more and more often in the United States, but not necessarily always and it needs to happen more often around the world as well. So, that’s a simple step that we can do to address the inequity for this cancer.

Are Sickle Cell Disease Patients More Susceptible to Renal Medullary Carcinoma?

This video was originally published by Diverse Health Hub here.

Are Sickle Cell Disease Patients More Susceptible to Renal Medullary Carcinoma? from Diverse Health Hub on Vimeo.

Are sickle cell disease patients more at risk for developing renal medullary carcinoma? In this highly informative interview, Dr. Pavlos Msaouel, a respected clinician at The University of MD Anderson Cancer Center specializing in treating RMC, shares why sickle cell disease patients and those with sickle cell trait may be more at risk for developing RMC.


Transcript:

Rebecca Law: 

Dr. Msaouel, are sickle cell disease patients more susceptible to renal medullary carcinoma?

Dr. Pavlos Msaouel:

So that is a wonderful question. So, it is not just individuals with sickle cell disease that are more susceptible to developing RMC, it also individuals with sickle cell trait. And there are many more individuals who have the sickle cell trait than those with disease.

Dr. Pavlos Msaouel:
About for every one individual with sickle cell disease there will be about 40 — that’s 4-0 — individuals with sickle cell trait. And many of them that carry the sickle cell trait will not know that they carry the sickle cell trait. So having either sickle cell disease or sickle cell trait increases substantially the risk for developing RMC. And why does that happen? It happens because when you carry either the sickle cell trait or sickle cell disease, your red blood cells — these are the cells in the blood that carry oxygen — normally, they will have this round shape, but if you have the sickle cell disease that shape is changed, it’s like a sickle, it changed to the form of a sickle.

Dr. Pavlos Msaouel:
If you have the sickle cell disease that will happen everywhere in your body. If you have the sickle cell trait, then your red blood cells will look normal in most areas of your body. However, there is one area in the kidney called the medulla of the kidney where the cells will look sickles whether you have sickle cell trait or sickle cell disease. And to our understanding, having these cells that look like sickles in the medulla damages the medulla in a way that increases the risk for developing this cancer. So RMC stands for renal medullary carcinoma and the medullary part in the middle of the name means that it comes from the medulla.

What is Renal Medullary Carcinoma?

This video was originally published by Diverse Health Hub here.

What is Renal Medullary Carcinoma? from Diverse Health Hub on Vimeo.

What is renal medullary carcinoma (RMC)? Dr. Pavlos Msaouel, a medical oncologist treating rare forms of renal cell and urothelial carcinomas, gives an overview of RMC, a rare, deadly type of kidney cancer predominantly afflicting young adults of African decent with sickle cell disease. Dr. Msaouel shares what symptoms have often led to diagnosis and points out that 75% of all RMC cases originate in the right kidney.


Transcript:

Rebecca Law:

Dr. Msaouel, what is renal medullary carcinoma and how is it diagnosed?

Pavlos Msaouel, MD, PhD:
RMC is the acronym for a kidney cancer named Renal Medullary Carcinoma. Renal Medullary Carcinoma or RMC is one of the deadliest kidney cancers, and it predominantly afflicts young people mainly of African descent that carry the sickle cell trait or sickle cell disease.

Pavlos Msaouel, MD, PhD:
It is often diagnosed after patients start developing symptoms, and those symptoms can be blood in the urine — the medical term is hematuria — or they may have pain in their flanks on their side where the kidneys would be, on the right side or on the left side.

Pavlos Msaouel, MD, PhD:
Interestingly RMC mainly happens on the right kidney, so about 75% of all RMC cases will happen in the right kidney. And unfortunately, in the vast majority of cases when it does present, it will be what we call stage 4 — meaning metastatic — meaning it will have spread to other organs.

Telemedicine: What You Need to Get Started

This post was originally published by Health Content Collective here.

Given the global pandemic, many healthcare providers are shifting gears to give patients the option to receive care virtually, without an in-person visit. The idea of telemedicine may seem a bit daunting so here’s what you need to know to help you have a smooth, effective experience.

What to Expect:

  • The Patient Portal: If you haven’t set this up in the past, now is a great time to get familiar with this technology. You can set up or manage your appointments, communicate with your doctor, view test results and access additional resources. It’s also where you’ll find the technology for virtual visits, if available from your provider. If you do not have login information, contact your provider’s office.

  • Apps: In some cases, the patient portal will have an app that serves the same purpose. For telemedicine visits, if available, this may be an easier-to-use option as it removes the nuances of compatibility with various browsers.

  • Instructions: Depending on your doctor’s office or institution, you will receive instructions for joining the virtual appointment. This may be in the form of an email message from the patient portal, a text message (if you’ve opted in), a phone call from the clinic, or all of the above.

  • Patience: As with any video chat technology, there is inevitably an issue early on either with sound, video or all of the above. Be patient with your provider and yourself as you try to connect. Conversation may not flow as easily as it does with your provider in person, but you will find a comfortable groove after the initial few minutes.

  • Consent: You will be asked to electronically sign and consent for the virtual appointment. You may receive a message from your doctor via the patient portal or you will be guided through the process as you log in for your visit.

Key Steps for Success:

  • Make sure you have access to the patient portal.
  • Login in advance to “test your system” – most technology offers this option when you are on the virtual appointment screen.

  • Download and login to the app if available.
  • When scheduling, ask for a phone number to call if you have any issues when connecting.

Will This Change the Practice of Medicine?

As we begin to look beyond the pandemic, it seems that virtual medicine may become a mainstay. Certainly, if your visit doesn’t require a physical exam, this option is enormously more convenient for seeing a doctor and being able to communicate how you’re feeling.  But if you need a test or bloodwork, this becomes a less reasonable option

What are we missing by visiting a doctor in-person? And, conversely, what are we gaining by going virtual? Like every new technology, there are pros and cons. Only time will tell.

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Managing Long Term Financial Concerns

This video was originally published by Cancer Support Community on April 29, 2015, here.

My Life Line

This resource was originally published by MyLifeLine.org here.

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The High Cost of Five Percent: The Importance of Capping Annual Out-of-Pocket Prescription Drug Costs for Medicare Part D Beneficiaries

This blog was originally published by Cancer Support Community by Kim Czubaruk.

Tuesday, June 25, 2019

Background on Medicare Part D

Approximately 43 million Americans are enrolled in Medicare Part D – a voluntary outpatient benefit provided through private health insurance plans approved by the federal government. These plans assist seniors in affording their prescription medications. However, for people with complex and serious health conditions who depend on high-cost drugs, affording medications under Medicare Part D is becoming more and more difficult. Below is a break-down of the different payment phases in Medicare Part D and an explanation of why imposing a cap (limit) on the amount beneficiaries pay out-of-pocket each year for prescription drugs is more important than ever.

The Different Payment Phases of Medicare Part D

While there are some differences between Part D plans, all Medicare Part D plans have the following:

  • A Monthly Premium – varies in cost by the plan selected (averaging $33.19/month in 2019) and must be timely paid each month to maintain coverage.
  • An Annual Deductible – varies in cost by the plan selected (but cannot exceed $415/year in 2019) and must be paid in full by the beneficiary before the Part D plan covers any costs of prescription drugs.
  • The Initial Coverage Phase – begins after the beneficiary has paid his or her annual deductible and triggers the Part D plan’s obligation to cover its share of the cost of the beneficiary’s prescription drugs.
  • Coverage Gap Phase (also known as the Doughnut Hole) – begins after the beneficiary and the Part D plan have paid a combined pre-determined amount for prescription drugs ($3820 in 2019) and triggers a phase where the beneficiary is obligated to pay the entire cost of his or her prescription drugs.
  • The Catastrophic Phase – begins after the beneficiary has paid a pre-determined amount in out-of-pocket costs ($5100 in 2019) for prescription drugs and triggers reduced beneficiary cost-sharing obligations for the remainder of the coverage period (in 2019, either 5% of the cost for each prescription medication, or $3.40 for each generic drug and $8.50 in for each brand-name drug, whichever is greater).

Catastrophic Phase of Medicare Part D

Thankfully, most Medicare Part D beneficiaries in 2019 will not have out-of-pocket prescription drug costs that exceed $5100. However, the high price of prescription drugs used to treat cancer and other serious medical conditions is causing more and more Medicare Part D beneficiaries to reach $5100 in out-of-pocket drug costs, sometimes early in the calendar year. When this happens, a beneficiary enters the Catastrophic Phase of Medicare Part D. What does this mean and why is it important?

  1. Once in the Catastrophic Phase, a beneficiary pays 5% of the cost for each prescription medication, or $3.40 for each generic drug and $8.50 for each brand name drug, whichever is greater;
  2. This payment obligation continues for the remainder of the calendar year;
  3. There is currently no cap or limit on the amount a beneficiary pays out-of-pocket annually in the Catastrophic Phase.

The 5% beneficiary cost-sharing in the Catastrophic Phase is the lowest cost-sharing percentage in Medicare Part D and is intended to minimize the cost burden on beneficiaries who have already incurred high out-of-pocket drug costs in any given calendar year. However, unlike commercial insurance plans, Medicare Part D does not cap or limit a beneficiary’s out-of-pocket prescription drug costs in a calendar year. Despite the good intentions behind the low 5% cost-sharing, the combined effect of high-priced prescription drugs and the absence of an out-of-pocket cap is making the Catastrophic Phase the costliest of all Medicare Part D Phases for an increasing number of beneficiaries.

Out-of-Pocket Cap in Medicare Part D

Creating an out-of-pocket cap for prescription drug costs in Medicare Part D would protect beneficiaries from unaffordable prescription drug prices and enable cancer patients and others confronting serious health conditions to focus on their health and take steps to avoid financial toxicity. There is draft legislation currently pending in the United States House of Representatives to cap out-of-pocket costs for Medicare Part D beneficiaries. The Cancer Support Community is encouraged by this development and will continue to monitor the progress of the draft legislation and voice the interests and concerns of cancer patients throughout the process.

To learn more about issues impacting cancer patients and engage in advocacy efforts, sign up to be a member of our Grassroots Advocacy Network.

Tips for Reducing Cost of Care

This video was originally published by Cancer Support Community on September 26, 2018, here.

 

 

 

 

Genomics and the Future of Cancer Treatment

This podcast was originally published by Cancer Care on October 15, 2018, here.

 

Topics Covered

  • Defining Genomics
  • The Role of the Pathologist
  • Microarrays & DNA Sequencing Technologies
  • The Role of Genomics in Your Treatment Choices
  • Family Cancer Syndromes
  • New Research on Genomics
  • Liquid Biopsies
  • Examples of How Genomics Help Identify Treatment Options
  • Key Questions to Ask Your Health Care Team about Genomic Testing and Its Benefits for You
  • Plans for Your Follow-Up Care
  • Questions for Our Panel of Experts

Our Panel of Experts

Raoul Tibes, MD, PhD

Director, Clinical Leukemia Program, Laura & Isaac Perlmutter Cancer Center; Associate Professor, NYU School of Medicine, Scholar in Clinical Research, Leukemia & Lymphoma Society, NYU Langone Health

Bob T. Li, MD, MPH

Medical Oncologist, Thoracic Oncology and Early Drug Development Service, Memorial Sloan Kettering Cancer Center

Sarah E. Kerr, MD

Consultant, Division of Anatomic Pathology and Laboratory Genetics and Genomics, Department of Laboratory Medicine and Pathology, Assistant Professor, Laboratory Medicine and Pathology, Director, Molecular Anatomic Pathology Laboratory, Co-Director, Genomics Laboratory, Mayo Clinic Cancer Center

Stewart B. Fleishman, MD

Former Founding Director, Cancer Support Services, Continuum Cancer Centers of New York, Author, Researcher in Oncology

Jessica M. Tarnowski, MGCS

Genetic Counselor, Department of Clinical Genomics, Mayo Clinic Cancer Center

Carolyn Messner, DSW, OSW-C, FAPOS, FAOSW

Director of Education and Training, CancerCare

Understanding Diagnostic Technologies and Biomarkers

This podcast was originally published by Cancer Care on May 6, 2015, here.

Topics Covered

 

  • Why the Molecular Portrait of Cancer is Important
  • The Role of the Pathologist
  • Benefits of Diagnostic Technologies, Biomarkers, Precision Medicine and Predicting Response to Treatment
  • Clinical Trials: How Research Contributes to Your Treatment Options
  • Questions to Ask Your Health Care Team
  • Quality-of-Life Concerns
  • Questions for Our Panel of Experts

 

Our Panel of Experts

Richard J. Gralla, MD, FACP

Professor of Medicine, Albert Einstein College of Medicine

Al B. Benson, III, MD, FACP, FASCO

Professor of Medicine, Associate Director for Clinical Investigations, Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Sarah E. Kerr, MD

Senior Associate Consultant, Division of Anatomic Pathology, Department of Laboratory Medicine and Pathology, Assistant Professor, Laboratory Medicine and Pathology, Mayo Clinic

Charles L. Loprinzi, MD

Regis Professor of Breast Cancer Research, Mayo Clinic College of Medicine

Caroline Edlund, MSW

Online Support Group Program Director, CancerCare

Prostate Imaging

This resource was originally published by the Prostate Cancer Research Institute here.

TRANSCRIPT:

“So your PSA number came back high. What now?”

Let’s say, like many men,  you’ve been getting your PSA checked every year as part of your routine checkup. In the past it has always hovered around 2 or maybe as high as 3.0, and suddenly it’s up to 5! Or maybe this is your first PSA test. Everyone knows that 4.0 is the magic number, anything above that means something is not right with your prostate. Right? Actually, it’s a lot more complicated than that, but for this video we’ll take that as a given. OK, so the number is high, what do you do? Well, if you’re like many men your first instinct might be something like this…

“Aaaah,  I’ve got prostate cancer! Cut the *****ing thing out”!

Unfortunately, there are a bunch of people in the medical community that will support and even encourage that instinct. It’s the wrong instinct.

Wait.

Slow down.

Take a deep breath.

Now just to be clear, this video is not advocating any sort of wonder-cure and we’re not downplaying how serious prostate cancer can be. Everything we’ll talk about here is backed up by good science, the latest technology, and highly trained medical professionals.

Let’s stop and talk for a second about the prostate. No offense to our creator, or to evolution or whatever you believe about human origins, but you couldn’t find a worse place to situate an organ, especially one that can be prone to problems. It’s…”down there”. And it’s in the middle of everything “down there”. Your urethra, that tube that carries urine out from the bladder, runs right through it… as do a couple of nerve bundles that control your ability to get and maintain an erection. And its located right in there close to the rectum. So, right in the middle of three really important systems.

OK. Back to your story. You’ve got the high PSA number. What’s likely to happen now?

Your general practitioner is probably going to want to refer you to a urologist. The urologist is almost certainly going to want to perform a needle biopsy and usually right away. It’s cancer! There’s urgency! Right?  Ummm. Not really. But we’ll get to that in a future video. For now, just know that for the overwhelming majority of men prostate cancer is very slow growing. You have time.

Here’s your first step in taking control. That’s what this is all about. Taking control of your own healthcare.

Unless your PSA number is crazy high (above 20 for example) or your GP has felt that something is wrong during the Digital Rectal Exam or DRE (that’s the finger up your butt exam that we all adore), you need to tell him or her that you want another PSA test. You see, that test measures a substance in the blood that the prostate gives off when it’s aggravated, and it can be aggravated by quite a few things other than cancer. Sexual activity, inflammation, certain types of heavy lifting, even riding a bike. So you’ll want to wait a few weeks, take it easy for the last few days, refrain from sex for 48 hours (you can do it), and retake the test. If your number has gone back down to near its normal level, you’re done for now. Just make sure to keep getting those yearly PSA tests and DREs.

If it remains high, then its time to move to the next step. Biopsy, right?

Nope. Not if you can help it.

The random needle biopsy, as it’s called, involves sticking a rather large needle into the area just in front of the rectum 12 times to remove samples, called cores, from different areas of the prostate.

It sounds horrible but, to be honest, it’s not terribly painful and it’s usually over in 10 or 15 minutes. A visit to the dental hygienist is probably just as uncomfortable. But it is invasive. Three percent of the men undergoing needle biopsies get infections, some of which are very serious. More importantly though, is the fact that the random needle biopsy is not very accurate. It can miss serious cancer or it can pick up low level, non-aggressive cancers that really don’t require treatment. (We’ll talk about the types of prostate cancer in a future video.)

The doctors who use the random needle biopsies don’t do it because they are mean or ignorant…maybe just a little slow to change. Until the last couple of years the needle biopsy was the best diagnostic tool that we had. Recent advances in MRI imaging have changed everything. The latest generation of MRI machines called 3 Tesla, or 3T machines, scan at a much higher resolution than the earlier machines. They enable radiologists to see all but the tiniest tumors. The tumors that they can’t see almost certainly don’t matter.

What improvements in imaging mean is that biopsies, when they are needed, can be targeted, right to the suspicious area in the prostate. No more random poking.

So, to summarize. If your PSA number comes back high:

Schedule a second PSA test.
Start doing some research. A good place to start is PCRI.org.
If the number is still high after that test, find an MRI center that does “Multi-parametric” testing using a 3T scanner. The MRI report will provide two types of important information:

The MRI measures the size of the prostate. The scan report will enable you to determine if your PSA elevation is proportionate to your prostate size. We’ll talk about this more in a future video. As regards cancer,  there are three possible outcomes:

No high-grade cancer. Further monitoring without biopsy is OK.
A high-grade lesion is detected.  Targeted biopsy is needed.
An ambiguous area is detected.  Another MRI in 6 months may be appropriate.

Scanning the prostate in men with PSA elevation is a brand new approach that is more reliable than the old-fashioned method of using 12 random needle sticks. However, this claim is only accurate when using the very latest state-of-the-art MRI technology at approved centers. This technology is so new that finding doctors willing to abandon the old random needle biopsy approach is still a major challenge.  Even so, there is a big payoff, being able to bypass those needles, those infections, and the inaccuracy is worth it.

Living with a Myeloproliferative Neoplasm

This video was originally published by Cancer Support Community on May 26, 2015, here.

 

First New Treatment in a Decade for MF Patients

This blog was originally published by PV Reporter on August 16, 2019, bDavid Wallace, here.

INREBIC provides new, once-daily oral option for patients affected by rare bone marrow cancer

SUMMIT, N.J.–(BUSINESS WIRE)–Celgene Corporation (NASDAQ: CELG) today announced the U.S. Food and Drug Administration (FDA) has approved INREBIC® (fedratinib) for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis.1

Celgene Fedratinib FDA approved for Myelofibrosis“The approval of INREBIC is another important milestone for Celgene and underscores our commitment to people living with blood cancers,” said Jay Backstrom, M.D., M.P.H., Chief Medical Officer for Celgene.  “We are excited to provide INREBIC as a new treatment option that may be used in patients with myelofibrosis, including patients previously treated with ruxolitinib.”

“Myelofibrosis can cause patients to suffer in many ways, including experiencing debilitating symptoms,” said Ruben Mesa, M.D., FACP, Director of the Mays Cancer Center at UT Health San Antonio Cancer Center MD Anderson.  “There has not been a new treatment approved for this disease in nearly a decade.  With INREBIC, physicians and patients now have another option available for myelofibrosis.”

The INREBIC development program consisted of multiple studies (including JAKARTA and JAKARTA2) in 608 patients who received more than one dose (ranging from 30 mg to 800 mg),1 of whom 459 had myelofibrosis,1 including 97 previously treated with ruxolitinib.1 The JAKARTA study evaluated the efficacy and safety of once-daily oral doses of INREBIC compared with placebo in patients with intermediate-2 or high-risk, primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis who were previously untreated with a JAK inhibitor, had enlarged spleens (a condition known as splenomegaly), and had a platelet count of ≥50 x 109/L (median baseline platelet count was 214 x 109/L; 16% <100 x 109/L and 84% ≥100 x 109/L).1,2 In the JAKARTA study, spleen volume was reduced by 35% or greater, when assessed from baseline to the end of cycle 6 (week 24), with a 4-week follow-up scan, in 37% (35 of 96) of patients treated with INREBIC 400 mg versus 1% (1 of 96) of patients who received placebo (p<0.0001).1 INREBIC also improved the Total Symptom Score as measured by the modified Myelofibrosis Symptoms Assessment Form (MFSAF) v2.0 diary2 (night sweats, itching, abdominal discomfort, early satiety, pain under ribs on left side, bone or muscle pain) by 50% or greater when assessed from baseline to the end of cycle 6 in 40% of (36 of 89) patients treated with 400 mg, versus 9% (7 of 81) of patients who received placebo (p<0.0001).1

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PV Reporter was the first to report Fedratinib by Celgene, offered on Expanded Access Program for Myelofibrosis patients in November, 2018.