Dr. John Mascarenhas shares advice for patients looking to learn more about clinical trials starting with consulting a specialist. Dr. Mascarenhas also emphasizes key questions to ask, including a discussion of the benefits versus risks of participating in a potential trial. 

Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

See More from Evolve Myelofibrosis

Related Resources:

Myelofibrosis Clinical Trial Participation | How Does It Move Research Forward?

Considering a Myelofibrosis Clinical Trial? Questions You Should Ask

Expert Perspective | A Concerted Effort to Advance Myelofibrosis Care

Transcript:

Katherine Banwell:

What about clinical trials? How can patients learn more? 

Dr. John Mascarenhas:

Well, clinical trials is definitely a confusing area because there are many clinical trials, and some of them are relevant to some patients may be not relevant to other patients.  

So, I think two ways is, see someone who does this. So, not everyone has as their primary care provider hematologist an expert, or someone who’s dedicated to doing this. They may be in a practice where they’re in the community setting; it’s not reasonable for them to be seen at a tertiary care center. 

But if you can get there even for a consultation or an initial visit to get plugged in, I think it’s really critical to see someone who is really invested in this with research opportunities, and clinical trial availabilities. And then the other resource would be clinicaltrials.gov.  

You can go in there, you can put in your diagnosis myelofibrosis. You can even manipulate it for geography to understand what trials are in your area. 

But it is a good way of looking, just to understand what’s there. And then the MPN Research Foundation, which is very supportive of the patient community and engages them, is often a very good resource to go to, to either learn about trials or join webinars where physicians that do this will discuss, and I think that’s another excellent resource. 

Katherine Banwell:

What questions should patients ask their team about clinical trials?   

Dr. John Mascarenhas:

Well, are there clinical trials for what I have? Because again, they can be very particular to where you are in the disease process, what medications you are on, what your kidney function is, things that may really influence decision-making. So, are there clinical trials, what are the clinical trials evaluating, what should I expect from them, both potentially from a positive angle, but also from a negative. What are the known toxicities, what would be the time commitment? Trials are more involved, and they’re more involved for a reason. 

I actually personally think patients do better on clinical trials than standard of care. The reason why I think that is because they are more involved. There’s a lot more oversight and eyes on you. Not just a physician perspective, but maybe more importantly, from a research nurse and research coordinator perspective. There’s a lot of regulatory burden which translates, I think, to a lot of attention to safety and assessment for advocacy.  

So, I think understanding what that looks like at any given institution, and how it will affect the patient and their caregivers from a time perspective, and obviously toxicity. But also, what is this trial trying to achieve? Does it make sense for what I’m trying to achieve? 

Katherine Banwell:

I suppose another question might be where this trial is taking place? As a myelofibrosis patient and a caregiver, are we going to have to travel to get to this clinical trial? 

Dr. John Mascarenhas:

I think that’s a major obstacle for a lot of patients. We’re talking about a disease that typically affects people that are in there sixth, seventh, eighth decade of life. Patients don’t all live, as I’ve said, right around a cancer center or a tertiary care center, so travel, the logistics of it, the ability to have that support available. Sometimes it’s loved ones, and adult children having to take time off of work to be able to help in that process.  

It’s a lot, and I’m particularly sensitive to it, because I work in a metropolitan area, and I realize getting in and out is not easy. And a lot of times, these trials understand that, and they build into the trials stipends and support for transportation and/or lodging which helps. It’s not perfect, but it can help at least financially, and sometimes logistically, so I would definitely inquire about what those resources are.  

And sometimes foundations also help bridge the gaps for patients to help link them to trials and facilitate that. So, it’s a super important part of engaging in a trial. 

Katherine Banwell:

And who is on the health care team that might be able to answer questions like this? 

Dr. John Mascarenhas:

Well, for sure, the physician should be able to. But I think the most valuable resource often is the nurse and the nurse practitioner. There are usually research coordinators. These are often young people, but very bright young people, that are very invested in this that will sometimes show up at the clinic to talk to the patient or will be a phone resource that you could reach out to. So, it can really be, I think, three levels. The coordinator, the nurse or nurse practitioner, or physician assistant, and the physician. So, it really shouldn’t be one person, but a team of people that are available to you.