All About REBLOZYL

What is REBLOZYL and how does it help to treat MDS?

REBLOZYL is in a erythroid maturating agent, which is a relatively new class of drug that was developed in the last few years, and it helps improve patients with MDS who have who are suffering with anemia and can improve their hemoglobin and decrease their need for red blood cell transfusions and just improve their quality of life.

Who is eligible for REBLOZYL and when is it typically used in MDS?

Patients with MDS who are suffering from anemia are generally eligible for REBLOZYL. Currently, we recommend REBLOZYL for patients who are requiring red blood cell transfusions. The initial approval for REBLOZYL was for patients with MDS that previously failed an erythropoietin stimulating agent, EPO, such as Aranesp or Procrit.

However, recent publications and research led to the approval of REBLOZYL in the front line setting for patients with MDS, and now they can.

Patients can take REBLOZYL whether they have failed an erythropoietin stimulating agent, or whether they are starting a first line agent to improve their anemia.

It's typically used in MDS for patients who are anemic and have red blood cell transfusion requirement.

How is REBLOZYL administered and what steps should patients follow before and after they receive it?

REBLOZYL is administered via subcutaneous injection. It's just a nurse will come in and give a shot in the arm or another location in the body.

It's very easy to administer. There are not a lot of steps that patients need to take before or afterwards. They might expect some pain with the injection like any other shot. Occasionally, patients will have a little redness or pain at the site afterwards as well, but it's usually extremely well tolerated.

What side effects could MDS patients experience while taking REBLOZYL?

And how are those side effects managed?

And the patients taking REBLOZYL may experience some side effects.

Fortunately, REBLOZYL is an extremely well tolerated drug. It's one of my favorite drugs to give, because it has such few side effects occasionally patients will experience fatigue. Sometimes it can get swelling in the legs. Low energy.

Very rarely, patients will have some nausea, shortness of breath.

Their blood pressure can go up. Some patients can also get a headache as well.

The main side effect that patients need to be aware of is that occasionally REBLOZYL can be so effective that at raises the hemoglobin very rapidly, which can lead to some dangerous side effects, but that's extremely rare and doesn't really happen with the way that we dose REBLOZYL currently.

How would a patient know that the drug is working for them?

Patients would know that REBLOZYL is working for them when they start to feel better, and when they start needing less red blood cell transfusions.

Fortunately, it's a quite effective drug. So a lot of patients are able to experience these good effects from REBLOZYL. And they hopefully can start experiencing these effects quite quickly.

How is REBLOZYL different from other treatments for MDS like chemotherapy or hypomethylating agents?

REBLOZYL is different from other MDS treatments such as hypomethylating agents or chemotherapy. And that it's whole goal is to try and improve the hemoglobin and try and prevent red blood cell transfusions.

Chemotherapy, is very, can be very toxic and difficult to tolerate for many patients. Hypomethylating agents or as I like to call light chemo can also be quite toxic.

Additionally, chemotherapy and hypomethylating agents can cause the platelets and white blood cells to go down as well.

REBLOZYL, on the other hand, really only affects the hemoglobin in terms of making it go up. Occasionally there is some patients might benefit in actually having their platelets and their neutrophils, their white blood cells go up as well.

This is some emerging data that we have right now.

Additionally REBLOZYL is only for patients currently with lower risk MDS as opposed to hypomethylating agents and chemotherapy which are normally given to patients with high risk MDS.

Occasionally, low risk MDS patients might get a hypomethylating agent, but that is usually for a very rare patient.

What are some of the common reasons why a patient with MDS might not be able to take REBLOZYL?

There would be very few reasons why a lower risk MDS patient might not be able to take REBLOZYL.

A few rare patients who have extremely high red blood cell transfusion requirements or have very severe bone marrow failure, where their white blood cell count is very low, extremely low, and where their platelets are extremely low might not really benefit from REBLOZYL. But most lower risk MDS patients who have anemia will benefit from REBLOZYL.

Are there any special instructions or precautions that MDS patients should be aware of or follow while they're using REBLOZYL?

For MDS patients, who are using REBLOZYL, they should follow what their doctor tells them.

This includes coming in for lab monitoring as needed, watching their blood pressure to make sure it doesn't get too high, monitoring their hemoglobin to make sure that the hemoglobin doesn't rise too fast, too quickly. They should also let their doctor know if they're experiencing any new side effects, in case that could be related to the REBLOZYL.

Otherwise, they should just follow normal standard precautions like they would with any new drug that they're taking.

What research or clinical trials are currently exploring REBLOZYL for MDS and how might that affect patients in the future?

There is a lot of research and clinical trials right now, exploring the use of REBLOZYL and MDS. Currently, we're actually I practice at Mount Sinai. We are participating in a clinical trial called the MAXILUS Trial, which is evaluating the use of REBLOZYL at the highest dose and then bringing that dose down if the patient needs. Current REBLOZYL dosing, you start at the lowest dose and then you work your way up to the highest dose

But we’re trying to see if you can get quicker responses in a safe manner starting at the highest dose. Additionally, there's other trials, evaluating REBLOZYL including the element trial, which is comparing REBLOZYL to erythropoietin stimulating agent in patients with low risk MDS who are not transfusion dependent. So currently REBLOZYL is only FDA approved for patients who are transfusion dependent. Additionally, there are combinations of REBLOZYL with other drugs.

There was a French group study that was, discussed at the American Society of Hematology meeting, last December in 2024, looking at the use of REBLOZYL in combination with erythropoietin stimulating agents in a certain subset of MDS patients.

Finally, REBLOZYL is being looked at with other drugs as well, including lenalidomide and there are other future clinical trials to come, with REBLOZYL combinations in lower risk MDS.

We hope that the these future clinical trials will further boost the efficacy of REBLOZYL, improve the outcomes and quality of life for a lower risk MDS patients.