What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndrome (MDS) include erythropoiesis-stimulating agents (ESAs), thrombopoietin mimetics, and immunomodulatory drugs like lenalidomide. ESAs can increase the risk of thromboembolic events and hypertension. Thrombopoietin mimetics, such as romiplostim and eltrombopag, may increase the risk of leukemic transformation and bone marrow fibrosis. Lenalidomide is associated with neutropenia, thrombocytopenia, pruritus, rash, and diarrhea. Statins, being studied for their anti-inflammatory and cardiovascular event prevention properties in MDS, are generally well-tolerated but can cause muscle pain, liver enzyme abnormalities, and an increased risk of diabetes.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically for the prevention of cardiovascular events or anti-inflammatory purposes in patients with Myelodysplastic Syndrome (MDS). However, treatments like erythropoiesis-stimulating agents (ESAs) and thrombopoietin receptor agonists (e.g., romiplostim and eltrombopag) have been explored for managing hematologic complications in MDS. These agents aim to improve blood cell counts and reduce the need for transfusions, although their use is associated with potential risks such as progression to acute myeloid leukemia (AML). Statins are being investigated in a pilot study for their potential to suppress myeloid malignancy progression and prevent cardiovascular events in lower-risk MDS patients.

What other types of research exist?

Promising novel alternatives to statins for MDS patients include investigational agents like imetelstat, an antisense oligonucleotide targeting telomerase, which has shown partial responses in advanced myelofibrosis and may offer anti-inflammatory benefits. Additionally, targeting specific mutations with agents like ivosidenib and enasidenib, which inhibit mutant forms of IDH1 and IDH2 respectively, could be considered, although their role in MDS is not well defined. These agents may provide therapeutic benefits while also potentially impacting inflammation and disease progression.

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HMG-CoA Reductase Inhibitor

Statins for Myelodysplastic Syndrome

Phase 2

Recruiting

Led By Amber Afzal, M.D., MSCI

Research Sponsored by Washington University School of Medicine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up pre-treatment, every 3 months while on treatment, end of treatment, 3 months after end of treatment and time of progression (estimated to be 15 months)

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing if statins, drugs usually used to lower cholesterol, can help patients with certain blood disorders by reducing inflammation and slowing disease progression. These patients currently have no effective treatments and are at risk of their condition worsening. Statins are commonly prescribed medications that have multiple beneficial effects.

Who is the study for?

This trial is for adults with CCUS or lower-risk MDS, which are conditions related to blood cell production. Participants must have specific genetic mutations and low levels of hemoglobin, white cells, or platelets. They should not be pregnant, breastfeeding, or have untreated HIV/HCV. Those who've taken statins in the last 6 months or other cancer treatments within a month can't join.

What is being tested?

The study tests whether Atorvastatin and Rosuvastatin can reduce inflammation and slow down genetic changes in patients with CCUS/MDS. It aims to see if these common cholesterol-lowering drugs could extend life by preventing disease progression and cardiovascular events.

What are the potential side effects?

Statins like Atorvastatin and Rosuvastatin may cause muscle pain, fatigue, digestive issues (like constipation), headaches, dizziness, increased blood sugar levels leading to diabetes risk, liver damage signs (e.g., yellowing skin/eyes), memory confusion or forgetfulness.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ pre-treatment, every 3 months while on treatment, end of treatment, 3 months after end of treatment and time of progression (estimated to be 15 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~pre-treatment, every 3 months while on treatment, end of treatment, 3 months after end of treatment and time of progression (estimated to be 15 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-treatment, every 3 months while on treatment, end of treatment, 3 months after end of treatment and time of progression (estimated to be 15 months) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change in allele burden (VAF) of somatic mutation

Side effects data

From 2014 Phase 3 trial • 58 Patients • NCT02084069

Postoperative bleeding

100%

80%

60%

40%

20%

Study treatment Arm

Treatment

Control

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: RosuvastatinExperimental Treatment1 Intervention

* Choice of statin is at the discretion of the treating physician and may depend on insurance approval. * Rosuvastatin dosing starts at 40 mg once daily. * In the absence of disease progression or intolerable side effects, patients may receive up to 12 months of treatment. * If a patient switches statins due to toxicity, treatment time is still limited to 12 months total (ie, if a patient receives 6 months of atorvastatin and switches to rosuvastatin, the duration of rosuvastatin will be no more than 6 months).

Group II: AtorvastatinExperimental Treatment1 Intervention

* Choice of statin is at the discretion of the treating physician and may depend on insurance approval. * Atorvastatin dosing starts at 80 mg once daily. * In the absence of disease progression or intolerable side effects, patients may receive up to 12 months of treatment. * If a patient switches statins due to toxicity, treatment time is still limited to 12 months total (ie, if a patient receives 6 months of atorvastatin and switches to rosuvastatin, the duration of rosuvastatin will be no more than 6 months).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Atorvastatin

1998

Completed Phase 4

~10900

Rosuvastatin

2019

Completed Phase 4

~3150

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Myelodysplastic Syndrome (MDS) include erythropoiesis-stimulating agents (ESAs), which stimulate red blood cell production to alleviate anemia, and thrombopoietin mimetics like romiplostim, which aim to increase platelet counts but carry risks of leukemic transformation. Statins, primarily used for cardiovascular event prevention, may also reduce inflammation and delay myeloid malignancy progression. Understanding these mechanisms helps tailor treatments to manage symptoms and improve survival outcomes for MDS patients.

Lenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes.Combination therapy with DNA methyltransferase inhibitors in hematologic malignancies.