Leukine

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Medically reviewed

Marina Burgos

Last updated on 10/01/2026

This page provides general, reference-level information compiled from official medical sources. It is not a substitute for professional medical advice, diagnosis, or treatment. For decisions about your health, please consult a qualified healthcare professional.

Overview of Leukine

Property Description
Active Ingredient Sargramostim
Form Sterile Solution for Injection, Lyophilized Powder for Reconstitution
Pharmacological Class Hematopoietic Growth Factor (Granulocyte-Macrophage Colony-Stimulating Factor, GM-CSF)
General Purpose Boosts the production and maturation of key white blood cells
Origin Recombinant Human Protein (Synthetic, yeast-derived)

Identity, Classification, and Purpose of Leukine

Leukine is the trade name for the pharmaceutical substance Sargramostim, which is medically classified as a Hematopoietic Growth Factor and a Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF). Its primary general purpose is to support the body's protective immune functions by stimulating the production of critical white blood cells.

Sargramostim is a recombinant human protein, meaning it is a synthetic, man-made version of a naturally occurring protein, produced using a specialized yeast expression system. This specific manufacturing process, which results in a glycosylated protein structure, is a key differentiating factor from other colony-stimulating factors. As a Colony Stimulating Factor (CSF), its core mechanism involves signaling the bone marrow to initiate the increase and maturation of immune cells, including neutrophils, monocytes, and macrophages. This action is clinically recognized for its role in cellular recovery and the restoration of these cell populations.

Composition and Available Forms of Sargramostim

The Leukine medicinal product contains Sargramostim as its single active ingredient. This medication is prepared for parenteral delivery—meaning it is given through injection or infusion—due to its protein nature, which would otherwise be broken down in the digestive system. The properties of Sargramostim align with a structure targeted toward hematopoietic cells.

Sargramostim is physically available either as a sterile, ready-to-use solution for injection or as a lyophilized powder that requires reconstitution prior to administration. The composition is stabilized within an aqueous solution to preserve the structural integrity of the active ingredient, ensuring the therapeutic protein reaches the bloodstream and mobilizes the body's resources for cellular defense. Leukine is designated as a prescription-only (Rx) medicine.

Regulatory References

  1. Sargramostim: MedlinePlus Drug Information

What side effects are possible with Leukine?

Possible Side Effects and Safety Information

The official safety profile for Leukine (sargramostim) is structured by classifying possible adverse reactions based on their frequency as observed in controlled clinical trials and the body system affected. This regulatory framework helps define the medicine's risk profile.


Frequency and System-Organ Classes

The most frequent events are classified as Very Common (occurring in ge 1 in 10 patients) and often involve the gastrointestinal and general systems. Specific reactions listed in regulatory documents include Diarrhea, Nausea, Vomiting, Fever (non-infectious), Stomatitis (mouth inflammation), Asthenia (weakness), Malaise, Rash, Edema, and Headache. Other affected system-organ classes include Musculoskeletal and Connective Tissue Disorders, presenting as Bone Pain and Myalgia.


Serious Adverse Reactions and Safety Constraints

The label identifies serious adverse reactions requiring attention. These include acute Hypersensitivity Reactions, such as anaphylaxis, as well as severe fluid and vascular events like Capillary Leak Syndrome and Supraventricular Arrhythmias (abnormal heart rhythm). Infusion-Related Reactions that may cause temporary respiratory distress or hypotension are also documented and are known to occur during or shortly after administration.

Safety constraints prohibit use in individuals with a known history of severe allergy to sargramostim or yeast-derived products. The label also advises against administering the medicine immediately before, during, or within 24 hours following cytotoxic chemotherapy or radiotherapy. Specific formulations containing benzyl alcohol are contraindicated for use in infants and neonates due to safety concerns in that population.

Overdose and Emergency Response

Overdose and when to seek help

Overdose with Leukine (sargramostim) is defined by the administration of doses exceeding the maximum recommended limit, leading to excessive pharmacological activity. The documented manifestations are primarily linked to the hematopoietic, cardiovascular, and vascular systems, and require immediate action as stated in official regulatory labeling.

Overdose scope Entity/Statement (Strictly from Regulatory Labeling)
Documented overdose presentations Dyspnea (shortness of breath), Nausea, Headache, Fever, Rash, and signs of Capillary Leak Syndrome (e.g., peripheral edema)
Severe outcomes Pericardial Effusion, potential for Cardiac Tamponade, and Excessive Leukocytosis (abnormally high white blood cell count)
Classification Statement (Strictly from Regulatory Labeling)
Antidote status No specific antidote is known for Sargramostim overdose.
Management Management is restricted to symptomatic and supportive treatment only.

In the event of suspected overdose or the manifestation of severe clinical signs, regulatory authorities mandate that emergency medical attention must be sought immediately. Specific triggers requiring urgent help include collapse, seizure, trouble breathing, or inability to be awakened. The mandated first step is the immediate discontinuation of Leukine therapy, followed by continuous hospital monitoring of complete blood counts and vital signs. If certain blood counts exceed official thresholds (e.g., white blood cell counts >50,000/ mm^3), administration must be interrupted or the dose reduced.

Therapeutic Uses of Leukine

Therapeutic Purpose of Leukine

Leukine (sargramostim) is a recombinant granulocyte-macrophage colony-stimulating factor (GM-CSF). Its primary function is to stimulate the bone marrow to produce white blood cells, specifically neutrophils, monocytes, and macrophages. These cells are essential components of the immune system that help the body protect itself against infections.

Main Uses

Leukine is utilized in several clinical scenarios where the production of white blood cells is compromised or needs to be accelerated:

Bone Marrow and Stem Cell Transplantation

In patients undergoing bone marrow transplantation (autologous or allogeneic), Leukine is used to reduce the time it takes for the new marrow to begin producing functional white blood cells. This process, known as myeloid engraftment, is critical for reducing the duration of vulnerability to severe infections. It is also used in cases where a bone marrow transplant has failed or is delayed in engraftment.

Peripheral Blood Progenitor Cell Mobilization

Leukine is used to mobilize hematopoietic progenitor cells from the bone marrow into the peripheral blood. This allows these cells to be collected and later infused back into the patient after they have received intensive chemotherapy.

Acute Myeloid Leukemia (AML)

In older adults diagnosed with acute myeloid leukemia, Leukine may be administered following induction chemotherapy. The goal is to shorten the period of severe neutropenia (low white blood cell count), thereby decreasing the risk of life-threatening infections during the recovery phase.

Radiation Exposure

Leukine is indicated to increase survival in patients who have been exposed to myelosuppressive doses of radiation. In these instances, it helps restore white blood cell production to mitigate the effects of acute radiation syndrome.

Benefits

The primary clinical benefits of Leukine therapy center on immune system support and recovery:

  • Reduction in Infection Risk: By accelerating the recovery of neutrophil counts, the period during which a patient is highly susceptible to bacterial and fungal infections is shortened.
  • Shortened Hospitalization: Faster immune recovery can lead to a reduction in the time patients need to remain in the hospital following intensive treatments or transplants.
  • Support for Intensive Therapy: By helping the bone marrow recover more quickly, Leukine allows patients to better tolerate the cycles of chemotherapy or radiation necessary for treating underlying conditions.

Eligibility and Restrictions for Use

Who Can and Cannot Use Leukine?

Eligibility for Leukine (sargramostim) is defined by regulatory authorities based on absolute prohibitions (contraindications), patient age, and pre-existing medical conditions.


Absolute Non-Eligibility (Contraindications)

Leukine is strictly contraindicated for use in patients with a known history of serious allergic reactions, including anaphylaxis, to sargramostim (GM-CSF), any other yeast-derived products, or any component of the Leukine formulation.


Age and Conditional Use Rules

Population Group Official Eligibility Status (Per Labeling)
Pediatric Patients Safety and efficacy are not established for most indications in children under 2 years of age. Approved for patients ge 2 years for post-transplant use.
AML Patients Approved for adults aged 55 years and older following induction chemotherapy. Safety has not been assessed in AML patients under 55.
Preexisting Conditions Must be used with caution in patients with a history of cardiac arrhythmia, congestive heart failure, fluid retention, or preexisting lung disease.

Pregnancy and Lactation

Leukine is not recommended during breastfeeding, and women are advised not to breastfeed during treatment. Use in pregnancy may cause fetal harm, and the benzyl alcohol–free formulation is recommended if treatment is necessary. Dosing must also be interrupted or reduced if white blood cell counts become excessively high.

What should I know about interactions with other medicines?

Interactions with other medicines and products

The officially documented interaction profile for Leukine (sargramostim) is defined by specific administration constraints and pharmacodynamic effects, rather than metabolic enzyme or transporter-mediated interactions. As a recombinant protein, it does not have a documented cytochrome P450 (CYP) interaction profile.

Administration-Timing Restrictions

Regulatory documents mandate strict timing separation with certain co-treatments. Leukine must not be administered simultaneously with, or within 24 hours preceding or 24 hours following, the completion of Cytotoxic Chemotherapy or Radiotherapy. This restriction is based on the requirement to avoid interference with treatments that target rapidly dividing cells.

Pharmacodynamic and Additive Effects

Co-administration with specific medicinal products may result in pharmacodynamic potentiation, meaning an additive effect on white blood cell production. The label notes that Lithium and Corticosteroids may potentiate the myeloproliferative effects of sargramostim.

Product and Population-Specific Notes

The official label does not document formal interactions with food, alcohol, or herbal products. A caution exists for the use of Leukine reconstituted with Bacteriostatic Water for Injection (containing benzyl alcohol) in neonates and low birth weight infants due to the presence of the excipient.

Mechanism of Action

GM-CSF Receptor Activation and Myelopoiesis

Leukine (sargramostim) is a recombinant Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) and functions as a selective agonist for the high-affinity GM-CSF receptor. This receptor is expressed primarily on hematopoietic progenitor cells in the bone marrow, as well as on mature cells of the myeloid lineage.

Binding to the receptor initiates intracellular signaling cascades, including the JAK/STAT and MAP kinase pathways. This molecular sequence drives the proliferation, differentiation, and survival of progenitor cells into committed circulating myeloid cells, specifically neutrophils and monocytes.

Modulation of Innate and Adaptive Immune Components

Beyond simple cell production, Leukine engages receptors on mature immune cells, enhancing their functional capacity. This results in increased rates of key physiological processes, including phagocytosis, cytotoxicity, and fungicidal activity, within neutrophils and macrophages. Furthermore, the mechanism promotes the differentiation and maturation of myeloid-derived dendritic cells (DCs), thereby influencing the early steps of the adaptive immune cascade.

Dosage and Administration Information

How to Use Leukine

The usage of Leukine (sargramostim) is defined by clinical guidelines concerning the method of administration, precise dose calculation, and the required treatment schedule. The medication is prepared for parenteral delivery, which means it is administered either as an intravenous (IV) infusion or a subcutaneous (SC) injection.


Official Administration Protocol

The standard prescribed adult dose for most indications, such as myeloid reconstitution following chemotherapy or transplantation, is 250 micrograms/ m^2/ day (micrograms per square meter per day), given once daily. For the IV route, the lyophilized powder must first be reconstituted and then diluted in 0.9% Sodium Chloride Injection before administration. To prevent drug loss, Albumin (Human) must be added if the final IV concentration is too low.

Administration is governed by a critical timing constraint: the medication must be initiated no sooner than 24 hours after the completion of cytotoxic chemotherapy or radiation therapy. Treatment continues daily until the Absolute Neutrophil Count (ANC) remains above 1500 cells/ mm^3 for three consecutive days.


Dosage Adjustments and Pediatric Use

Usage instructions incorporate a mandatory dose adjustment based on lab results: the dose must be interrupted or reduced by 50% if the ANC exceeds 20,000 cells/ mm^3. For pediatric patients, the dose is typically calculated based on body surface area, applying the 250 micrograms/ m^2/ day dose to those two years of age and older in the BMT setting. In cases of Acute Radiation Syndrome (H-ARS), a weight-based dose of 7 micrograms/ kg/ day SC is prescribed for adults over 40 kg, with specific lower mcg/ kg amounts for children based on weight tiers.

Recent Clinical Evidence

Research evidence / Overview of Studies for Leukine (Sargramostim)

This overview summarizes the research evidence and study landscape for Leukine (sargramostim), focusing on the types of trials and research studies used by health authorities to evaluate its purpose.


Evidence for Use in Myeloid Recovery Following Bone Marrow Transplantation (BMT) Procedures

The core evidence for Leukine was studied in the post-BMT setting comes from randomized, controlled clinical trials. These studies were used in research examining temporary physiological imbalance in patients who had received either an autologous or allogeneic bone marrow transplant. Researchers monitored outcomes related to systemic or functional imbalance, such as the time it took for white blood cell counts (neutrophils) to recover and the overall duration of the patient's hospitalization following the procedure. Studies explored the impact on the frequency of acute or disruptive episodes, such as severe infections or bacteremia, across groups receiving Leukine compared to groups receiving an inactive substance (placebo). Findings describe patterns observed in the studies related to the measured time for neutrophil recovery across the groups. Limited information for long-term outcomes exists, particularly concerning the durability of the response years after the initial treatment.


Evidence for Use in Myeloid Recovery After Acute Myeloid Leukemia (AML) Chemotherapy

Research was conducted during periods of increased symptom activity following intensive chemotherapy for newly diagnosed Acute Myeloid Leukemia. The most significant data comes from Phase 3 randomized, double-blind, placebo-controlled trials. These studies focused primarily on adult patients aged 55 and older. Studies monitored outcomes related to physical discomfort and systemic strain, specifically by measuring the time until white blood cell counts reached specific thresholds. Research examined the incidence of severe and life-threatening infections in the observed populations. The primary evidence is confined to the specific populations studied, meaning that the results apply only to the older adults and the specific chemotherapy regimens that were used in the trials. Long-term effects are not fully established regarding disease remission or overall survival.


Research Gaps and Areas of Uncertainty

The scientific literature highlights several areas where more research is needed. Comparative evidence is lacking in certain scenarios, meaning that direct, randomized comparisons of Leukine against all other available treatment options are not universally available across all approved indications. Findings indicated variability when Leukine was evaluated in studies exploring the mobilization of blood stem cells for collection. Furthermore, subgroup findings are uncertain for patients with specific comorbid conditions or disease subtypes, as the results apply only to the populations studied in the main trials.

Frequently Asked Questions (FAQ)

Common questions about Leukine (FAQ)


Q: What is the main difference between Leukine and other colony-stimulating factors?

Leukine (sargramostim) is officially classified as a Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF). This means it stimulates the growth of a broad group of immune cells, including granulocytes and macrophages. Furthermore, it is a recombinant human protein produced in a specific yeast expression system, which is a differentiating characteristic from other common colony-stimulating factor medicines.

Q: Can Leukine be used for any condition besides cancer treatment?

Yes. Beyond its uses related to specific chemotherapy and transplantation procedures, official regulatory documents indicate that Leukine is also approved for increasing survival in adults and children acutely exposed to myelosuppressive doses of radiation, a condition known as Acute Radiation Syndrome (H-ARS).

Q: What is the role of white blood cells when taking Leukine?

Leukine's primary function is to stimulate the bone marrow to produce key white blood cells like neutrophils and monocytes. The official mechanism describes how it enhances the function of mature immune cells, including increasing their ability to perform phagocytosis (engulfing foreign particles) and cytotoxicity (cell-killing activity), processes that are critical to the body's protective functions.

Q: Is Leukine safe to use for patients with a known heart condition?

Official labeling advises that Leukine should be used with caution in patients who have a history of certain pre-existing heart conditions, specifically cardiac arrhythmia (abnormal heart rhythm) or congestive heart failure. Due to this cautionary note, regulatory guidelines require patients in these groups to be carefully monitored by the healthcare team during treatment.

Q: Can older adults use Leukine without increased risk?

The use of Leukine is approved for specific indications in older adults, such as those aged 55 years and older with Acute Myeloid Leukemia. Regulatory documents note that in the specific trials reviewed, appropriate studies performed to date did not demonstrate geriatric-specific problems that would limit the usefulness of the medicine in the studied populations.

Q: Does alcohol interact negatively with Leukine?

According to the official product information, the regulatory label does not document a formal interaction between Leukine and alcohol. A healthcare professional remains the resource for specific guidance on alcohol consumption during treatment.

Q: Can Leukine be taken alongside herbal supplements?

The official product information does not document formal interactions between Leukine and herbal products. All herbal supplements are advised to be reviewed with a healthcare professional before they are taken during treatment.

Q: What should I tell my doctor before starting Leukine?

Regulatory guidance highlights several conditions and factors that must be shared with your doctor before starting treatment. These include any history of serious allergies to sargramostim or yeast products, pre-existing health issues like heart arrhythmia or lung disease, and whether you are or plan to become pregnant or are breastfeeding.

Q: Is it possible to be allergic to Leukine?

Yes, it is possible. Leukine is strictly contraindicated (not allowed) in patients with a known history of a serious allergic reaction, such as anaphylaxis, to sargramostim or yeast-derived products. Acute Hypersensitivity Reactions are also listed as a serious possible adverse reaction in the official safety profile.

Q: Can Leukine cause a reaction at the injection site?

While the official label lists general skin side effects like Rash and Edema (swelling) as common adverse reactions, specific data on localized injection-site pain or redness is not provided among the most frequent events. Infusion-related reactions that may cause temporary breathing difficulty or low blood pressure are documented to occur during or shortly after administration.

Q: Is Leukine a type of chemotherapy drug?

No. Leukine is classified as a Hematopoietic Growth Factor. It is a protein treatment used to stimulate the recovery of certain white blood cells following treatments like chemotherapy or radiation therapy, which can lower blood cell counts.

Q: Is Leukine the same as Neulasta or Neupogen?

No, Leukine is not the same. Leukine is classified as a GM-CSF (Granulocyte-Macrophage Colony-Stimulating Factor), which stimulates the growth of both neutrophils and monocytes. Other similar drugs, such as Neupogen or Neulasta, are classified as G-CSF, which primarily focuses on stimulating the growth of only neutrophils.

Q: How quickly does Leukine start working after the first dose?

The official product information describes the drug's action on blood cells as rapid, indicating that the process of cell production begins quickly after administration. However, the regulatory label does not specify an exact number of hours for the onset of the clinical effect.

Q: How long do the effects of Leukine last in the body?

The drug's elimination time is reported in official regulatory documents. Its activity is characterized by a terminal half-life—the time it takes for half of the substance to be eliminated—of approximately 1.7 hours following intravenous infusion.

Q: Do I need to change my diet while taking Leukine?

The official label does not document any formal interactions between Leukine and food. Therefore, no specific requirement for general dietary changes is specified in the regulatory information.

Q: Are there any common over-the-counter medications that interact with Leukine?

The official interactions section primarily identifies other treatments like chemotherapy, radiotherapy, Lithium, and Corticosteroids. Common over-the-counter medications are not listed as having formal interactions, but a healthcare provider remains the resource for specific guidance before taking any additional medicine.

Q: Is it normal to feel tired after receiving Leukine?

A general feeling of Asthenia (weakness) and Malaise (general discomfort) are listed as very common adverse reactions in the official safety profile. This means these effects were reported by 1 in 10 or more patients in clinical trials.

Q: Does Leukine make a person more susceptible to infections?

No. Leukine is used to stimulate immune cell recovery and its approved uses involve reducing the time of neutrophil recovery in clinical trials. Its mechanism enhances immune cell functions, such as phagocytosis, which is intended to support the body’s protective mechanisms.

Q: Are there any restrictions on travel while undergoing Leukine treatment?

The main restriction involves the need for refrigeration. Unopened vials must be stored under refrigeration between 2 C and 8 C (36 F and 46 F) and must not freeze. If traveling, specific measures must be taken to ensure these temperature requirements are maintained.

Q: Are there long-term side effects associated with Leukine use?

Official regulatory summaries of the clinical studies indicate that limited information is available for long-term outcomes and the durability of the response years after the initial treatment. Therefore, the long-term effects of the medicine are not fully established.

Q: What happens when treatment with Leukine is stopped?

Treatment is typically stopped when the white blood cell count (ANC) reaches a specific target level for three consecutive days. The official label requires that blood counts continue to be monitored for a period after the medication is discontinued to ensure stable cell recovery.

Q: Why is Leukine not recommended for everyone with low white blood cells?

Leukine is strictly contraindicated (not allowed) for use in patients with a known history of serious allergies to sargramostim, yeast-derived products, or any component of the formulation, regardless of their blood cell count. These contraindications are in place to ensure patient safety.

Q: Does Leukine affect blood pressure?

Yes. The official label documents that hypotension (low blood pressure) can occur as part of a documented infusion-related reaction. These reactions may occur during or shortly after administration and require careful monitoring.

Q: Does Leukine cause hair loss?

Hair loss (alopecia) is not listed among the Very Common or Common adverse reactions (occurring in 1 in 10 or 1 in 100 patients) detailed in the official label's summary of clinical trial experience.

Q: Is Leukine a biologic medicine?

Yes. Leukine (sargramostim) is described in regulatory documents as a recombinant human protein produced by specialized DNA technology. This classification is consistent with the definition of a biologic medicine.

Q: Is Leukine a vaccine?

No. Leukine is medically classified as a Hematopoietic Growth Factor. It is a treatment used to stimulate the production of certain blood cells in the bone marrow, not a vaccine designed to create active immunity against a disease.

Q: Can Leukine make certain medical tests inaccurate?

Yes. The drug's intended action is to cause a significant increase in white blood cell counts (leukocytosis). This change in blood cell levels is a direct effect of the drug that will alter the results of common tests like a complete blood count (CBC), requiring mandatory monitoring and dose adjustment.

Q: Does Leukine only affect white blood cells?

The primary effect of Leukine is on the white blood cell line, specifically driving the proliferation and differentiation of cells into neutrophils and monocytes. This is the focus of its classification as a Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF).

Q: Is Leukine used to treat low platelet counts?

Leukine's primary mechanism of action is focused on stimulating the production and differentiation of myeloid cells, such as neutrophils and monocytes. Although it is not the primary target, the label does require monitoring platelet counts during the course of treatment.

How should Leukine be stored and disposed of?

How to Store and Dispose of Leukine (Sargramostim)

Official regulatory guidelines strictly define the conditions required to maintain the stability and safety of Leukine.

Storage and Handling Requirements

Condition Requirement
Temperature Store unopened vials under refrigeration at 2 C to 8 C (36 F to 46 F). Do not freeze.
Handling Do not shake the vial. Keep out of direct sunlight.
Stability After reconstitution, storage time depends on the diluent: 20 days if mixed with Bacteriostatic Water, or 24 hours if mixed with Sterile Water.
Child Safety Keep all medication out of the sight and reach of children.

Disposal Instructions

Used needles and syringes must be placed in a designated, puncture-proof sharps container. Unused or expired medication should not be disposed of in household trash or poured down the toilet or sink, but should be returned to a pharmacy or collection point as directed by a healthcare provider.

Attention! Always consult to a doctor or pharmacist before using pills or medicines.

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