Sargramostim for Blood Cancer

Overseen By Stacey Brown, BA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Northside Hospital, Inc.

Disqualifiers: Poor organ function, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately 3 weeks

38 Participants Needed

Trial Summary

What is the purpose of this trial?

Given the increased number of HLA-mismatched haploidentical transplantation with post-transplant cyclophosphamide performed each year and the high risk of infectious complications associated with this type of transplant, the investigators suggest that GM-CSF administration post-infusion of T-replete haploidentical stem cells and post-transplant cyclophosphamide can yield similar count recovery rates to G-CSF with a potential of lowering risk of infectious complications.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Sargramostim for blood cancer?

Sargramostim, also known as rhu GM-CSF, is used to help the body recover after bone marrow transplants and chemotherapy by boosting the immune system. It has been shown to improve immune recovery and increase certain immune cells, which can be beneficial in cancer treatment. ^{1 2 3 4 5}

Is Sargramostim safe for humans?

Sargramostim, also known as rhu GM-CSF, has been used since 1991 to help recover bone marrow after chemotherapy and is generally considered safe, but some people have reported allergic reactions. A study comparing it to another similar drug found it to be well-tolerated, with only a slightly higher chance of mild fever. ^{1 2 4 5 6}

How is the drug sargramostim unique for treating blood cancer?

Sargramostim is unique because it boosts the immune system by increasing the number of certain immune cells, which can help the body recover from chemotherapy and fight infections. Unlike some other treatments, it is derived from yeast and is used to speed up recovery of bone marrow and enhance immune responses. ^{1 2 3 4 5}

Research Team

Melhem Solh, MD

Principal Investigator

Northside Hospital

Eligibility Criteria

This trial is for patients with various blood cancers who need a stem cell transplant and have a partially matched related donor. They must be in good physical shape (KPS >/= 70%) and able to consent. It's not for those with poor heart, lung, liver, or kidney function, HIV-positive individuals, or anyone allergic to GM-CSF or yeast products.

Inclusion Criteria

I have a blood disorder that qualifies for a bone marrow transplant.

You need to have a family member who is a good match for a transplant (with a matching score of 5/10 to 8/10).

I am mostly able to care for myself.

Exclusion Criteria

History of severe or serious allergic reaction to human GM-CSF or yeast-derived products

HIV-positive

You have a serious medical or mental condition that would prevent you from understanding and agreeing to participate in the study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Sargramostim (GM-CSF) starting on Day +5 post-transplant until ANC >1000 x3 days or >1500 x1 day, administered not less than 24 hours after the last dose of cyclophosphamide

Approximately 3 weeks

Daily visits for infusion administration

Follow-up

Participants are monitored for safety, engraftment, and infection rates post-treatment

3 months

Regular visits for monitoring and assessments

Long-term follow-up

Participants are monitored for relapse rates, GVHD, and overall survival

12 months

Treatment Details

Interventions

• Sargramostim

Trial Overview The study tests if GM-CSF can help recover blood counts after a haploidentical stem cell transplant with post-transplant cyclophosphamide treatment while reducing infection risks compared to the standard G-CSF approach.

Participant Groups

Group I: GM-CSF post-transplant Experimental Treatment 2 Interventions

Sargramostim (GM-CSF) will start on Day +5 and continue until ANC \>1000 x3 days or \>1500 x1 day. GM-CSF will be administered not less than 24 hours after the last dose of cyclophosphamide and will be given at a dose of 250mcg/m2/day as an infusion over 2 hours.

Who Is Running the Clinical Trial?

Northside Hospital, Inc.

Lead Sponsor

Trials

26

Recruited

1,100+

Findings from Research

In a study of 15 pediatric patients with malignancies and invasive fungal diseases (IFDs), sargramostim showed a high overall response rate of 92%, indicating its potential effectiveness as an adjunctive treatment for patients who are neutropenic and refractory to antifungal therapy.

A systematic review of 65 cases, including the 15 new patients, demonstrated an overall response rate of 82% for sargramostim in treating IFDs, suggesting it may serve as a valuable immunomodulator for patients with hematological malignancies facing difficult-to-treat fungal infections.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases. Chen, TK., Batra, JS., Michalik, DE., et al.[2022]

A case report described an adverse reaction to sargramostim (rhu GM-CSF) involving symptoms like itching, hives, and throat tightness, highlighting the potential for allergic reactions to this treatment.

Prick skin testing showed that the patient was sensitized to sargramostim but not to filgrastim (rhu G-CSF), suggesting that skin testing could help identify patients at risk for allergic reactions to GM-CSF therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immediate hypersensitivity to human recombinant granulocyte-macrophage colony-stimulating factor associated with a positive prick skin test reaction. Engler, RJ., Weiss, RB.[2017]

In a study involving 35 patients (18 receiving GM-CSF and 17 controls), GM-CSF did not enhance T cell or natural killer cell recovery after allogeneic stem cell transplantation, contrary to expectations.

However, GM-CSF administration improved dendritic cell reconstitution in patients undergoing autologous stem cell transplantation, suggesting its benefits may vary based on the type of transplant.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Granulocyte-macrophage colony-stimulating factor increases the proportion of circulating dendritic cells after autologous but not after allogeneic hematopoietic stem cell transplantation. Eksioglu, EA., Kielbasa, J., Eisen, S., et al.[2018]

In a study involving 137 cancer patients undergoing chemotherapy, both sargramostim and filgrastim were found to be well tolerated for preventing chemotherapy-induced neutropenia, with only a slightly higher incidence of mild fever associated with sargramostim.

There were no significant differences in adverse events, hospitalization days, or the need for intravenous antibiotics between the two treatments, indicating that both growth factors are comparably effective in this context.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized trial comparing the tolerability of sargramostim (yeast-derived RhuGM-CSF) and filgrastim (bacteria-derived RhuG-CSF) in cancer patients receiving myelosuppressive chemotherapy. Beveridge, RA., Miller, JA., Kales, AN., et al.[2019]

Sargramostim is effective in accelerating hematologic recovery in patients exposed to bone marrow-suppressing agents, leading to fewer infections and improved survival outcomes in various clinical settings.

As an immune modulator, sargramostim enhances anti-cancer responses when used alongside conventional therapies, showing a favorable safety profile and potential for expanded use in treating immune-related conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sargramostim (rhu GM-CSF) as Cancer Therapy (Systematic Review) and An Immunomodulator. A Drug Before Its Time? Lazarus, HM., Ragsdale, CE., Gale, RP., et al.[2022]

In a study analyzing 990 patients treated with sargramostim and matched cohorts receiving filgrastim or pegfilgrastim, sargramostim was associated with a 56% lower risk of infection-related hospitalizations for those undergoing chemotherapy-induced neutropenia.

Patients using sargramostim also experienced significantly lower hospitalization costs, with reductions of 84% compared to filgrastim and 62% compared to pegfilgrastim, highlighting its potential economic benefits in clinical settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of hospitalization risk and associated costs among patients receiving sargramostim, filgrastim, and pegfilgrastim for chemotherapy-induced neutropenia. Heaney, ML., Toy, EL., Vekeman, F., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Immediate hypersensitivity to human recombinant granulocyte-macrophage colony-stimulating factor associated with a positive prick skin test reaction. [2017]

3. England pubmed.ncbi.nlm.nih.gov

Granulocyte-macrophage colony-stimulating factor increases the proportion of circulating dendritic cells after autologous but not after allogeneic hematopoietic stem cell transplantation. [2018]

4. Germany pubmed.ncbi.nlm.nih.gov

Randomized trial comparing the tolerability of sargramostim (yeast-derived RhuGM-CSF) and filgrastim (bacteria-derived RhuG-CSF) in cancer patients receiving myelosuppressive chemotherapy. [2019]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Sargramostim (rhu GM-CSF) as Cancer Therapy (Systematic Review) and An Immunomodulator. A Drug Before Its Time? [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Comparison of hospitalization risk and associated costs among patients receiving sargramostim, filgrastim, and pegfilgrastim for chemotherapy-induced neutropenia. [2022]