What side effects are associated with this type of intervention?

The common treatments for Prolymphocytic Leukemia, particularly those involving Post-Transplant Cyclophosphamide (PTCy), Sirolimus, and Mycophenolate Mofetil (MMF) for GVHD prophylaxis, have several known side effects. Cyclophosphamide can cause nausea, vomiting, hair loss, and an increased risk of infections due to bone marrow suppression. Sirolimus may lead to mouth sores, high blood pressure, elevated cholesterol levels, and impaired wound healing. Mycophenolate Mofetil (MMF) is associated with gastrointestinal disturbances, such as diarrhea and abdominal pain, as well as an increased risk of infections. These side effects are important considerations for patients undergoing treatment and should be discussed with their healthcare provider.

What prior FDA approvals exist?

The FDA has approved several treatments for Prolymphocytic Leukemia (PLL), including chemotherapeutic agents like alemtuzumab and pentostatin. While these treatments differ from the combination of Post-Transplant Cyclophosphamide (PTCy), Sirolimus, and Mycophenolate Mofetil (MMF) used for GVHD prophylaxis in stem cell transplantation, they share a common goal of targeting rapidly proliferating cells and modulating the immune response. Alemtuzumab, for instance, is a monoclonal antibody that depletes T-cells, similar to how PTCy and Sirolimus work to prevent graft-versus-host disease by modulating immune activity.

What other types of research exist?

Promising novel alternatives for GVHD prophylaxis in Prolymphocytic Leukemia patients include the use of JAK inhibitors like ruxolitinib, which has shown efficacy in reducing GVHD severity. Additionally, abatacept, a CTLA-4 Ig fusion protein, has demonstrated potential in preventing acute GVHD. Another alternative is the use of vedolizumab, an anti-integrin antibody, which targets gut-homing T cells and may reduce gastrointestinal GVHD. These alternatives are being actively studied and may offer new options for patients in 2024.

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Other

Stem Cell Transplant + Chemo for Blood Cancer

Phase 2

Recruiting

Led By Mark Juckett

Research Sponsored by Masonic Cancer Center, University of Minnesota

Eligibility Criteria Checklist

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

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 72 months

Awards & highlights

No Placebo-Only Group

Summary

This trial involves preparing patients with drugs and radiation before giving them a donor stem cell transplant. After the transplant, medications are used to prevent rejection of the new cells. It targets patients who need stem cell transplants.

Who is the study for?

This trial is for people aged 0-75 with various blood diseases or leukemias in remission, who have a compatible donor for stem cell transplant. They must not be pregnant, breastfeeding, or have untreated infections and should have adequate heart, lung, liver, and kidney function. HIV+ individuals can join if they're on treatment with an undetectable viral load.

What is being tested?

The study tests a non-myeloablative preparative regimen using cyclophosphamide/fludarabine/total body irradiation followed by stem cell infusion from related/unrelated donors. Post-transplant care includes cyclophosphamide (PTCy), sirolimus and mycophenolate mofetil to prevent graft-versus-host disease.

What are the potential side effects?

Possible side effects include reactions to medication infusions; organ damage due to the drugs or radiation; increased risk of infections; graft-versus-host disease where the new cells attack the body; and complications from suppressed bone marrow function.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 72 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~72 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 72 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Evaluate rates of acute graft-versus-host disease (GVHD)

Evaluate rates of chronic graft-versus-host disease (GVHD)

Secondary study objectives

Observe rates of relapse (RR)

Observe transplant related mortality (TRM)

Overall Survival (OS)

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Cy/Flu/TBI + Post transplant CYExperimental Treatment8 Interventions

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mycophenolate Mofetil

1997

Completed Phase 4

~2380

Peripheral Blood Stem Cell Transplant

2011

Completed Phase 2

~80

Allopurinol 300 MG

2019

Completed Phase 3

~700

Fludarabine

2012

Completed Phase 4

~1860

Cyclophosphamide

2010

Completed Phase 4

~2310

Total Body Irradiation

2006

Completed Phase 3

~820

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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

The most common treatments for Prolymphocytic Leukemia (PLL) include Cyclophosphamide, Sirolimus, and Mycophenolate Mofetil (MMF). Cyclophosphamide works by cross-linking DNA strands, leading to cell death, which is essential for targeting rapidly dividing leukemia cells. Sirolimus inhibits mTOR, preventing T-cell activation and proliferation, thus reducing the risk of graft-versus-host disease (GVHD) post-transplant. MMF inhibits inosine monophosphate dehydrogenase, crucial for purine synthesis in lymphocytes, thereby suppressing the immune response and further preventing GVHD. These mechanisms are critical for PLL patients as they help manage the disease and prevent post-transplant complications.

Selective restoration of immunosuppressive effect of cytotoxic agents by thymopoietin fragments.Unexpected remission of acute myeloid leukaemia after GM-CSF.Rational therapeutic options for patients with myeloproliferative neoplasms.