What Is Palifermin? Definition and What Research Reports

Definition

Palifermin is a recombinant human keratinocyte growth factor (rHuKGF, also called KGF-1), a laboratory-produced, N-terminally truncated form of the naturally occurring human protein fibroblast growth factor 7. It is expressed in Escherichia coli rather than extracted from human tissue, and it acts on the keratinocyte growth factor receptor found on epithelial cells lining the mouth, throat and gastrointestinal tract. In clinical research it has been administered intravenously and studied chiefly as a way to reduce the severity and duration of oral mucositis — the painful breakdown of the mouth and throat lining — in people receiving high-dose chemotherapy and total body irradiation before haematopoietic stem cell transplantation, an indication summarised in drug reviews of palifermin (PMID 16225371, PMID 17059384).

This page is for educational purposes only and is not medical advice; consult a licensed physician for any health decision. Nothing here describes how palifermin should be used.

Molecule Class and Origin

Palifermin belongs to the growth factor class of therapeutic proteins rather than to the short synthetic peptide category. It is a single-chain polypeptide of roughly 140 amino acids, shorter than native KGF, a modification that reviewers described as improving stability while retaining receptor binding activity (PMID 16225371). Because the keratinocyte growth factor receptor is expressed on epithelial cells but not on cells of haematopoietic lineage, reviewers described the proposed mechanism as selective stimulation of epithelial proliferation and differentiation in mucosal tissue (PMID 17190850).

Why it appears in peptide glossaries

Palifermin is frequently listed alongside peptides in reference material because it is a recombinant protein drug whose activity depends on a defined amino acid sequence binding a specific receptor — the same conceptual framework used to describe smaller research peptides. It is not a research chemical: it is a regulated biologic product with published pharmacology, and the literature discussed here comes from clinical trials, pharmacokinetic studies, animal models and published case reports.

How the Term Is Used in the Literature

Across the published record, "palifermin" is used interchangeably with "recombinant human keratinocyte growth factor" and "rHuKGF". Three usage contexts dominate:

• Mucositis prevention research — the largest body of work, centred on haematological malignancy and stem cell transplantation settings, described in a core-evidence review of palifermin for oral mucositis (PMID 20694076).
• Pharmacology studies — healthy-volunteer work characterising absorption, clearance and biomarker responses (PMID 16765144).
• Nursing and supportive-care literature — papers framing palifermin as a shift from treating mucositis symptoms toward preventing the lesion, as one European nursing review described (PMID 17540295).

What the Published Literature Reports

Preclinical work

Animal research preceded the clinical programme. Researchers in a mouse model reported that recombinant human keratinocyte growth factor reduced radiochemotherapy-induced early oral mucositis in irradiated oral mucosa (PMID 15936573). That experimental work established the epithelial-protection rationale later tested in humans.

Clinical mucositis outcomes

A core-evidence review of palifermin concluded that the agent ameliorated oral mucositis in patients with haematological malignancies undergoing myelotoxic therapy (PMID 20694076). An expert-opinion review similarly described palifermin as a keratinocyte growth factor that reduced oral mucositis after stem cell transplant for haematological malignancies (PMID 17059384). A separate review examined current evidence and future perspectives for palifermin in the management of mucositis in haematological malignancies, including open questions about broader tumour settings (PMID 19607642). Researchers also evaluated palifermin in children undergoing autologous stem cell transplantation using a matched-pair analysis design (PMID 25503176).

Pharmacokinetics and interactions

A healthy-volunteer study assessed the pharmacokinetics, pharmacodynamics and safety of palifermin (rHuKGF) outside the oncology setting, characterising how the protein behaved in people without mucosal injury (PMID 16765144). Later investigators examined pharmacokinetic and pharmacodynamic interactions between palifermin and heparin, a question that arises because heparin is commonly present in intravenous access lines (PMID 25880826).

Adverse Events: What Studies Report

The published safety record includes dermatological and mucosal findings. Dermatologists reported a palifermin-associated papular eruption in a published case description (PMID 19221263). A separate clinical and histological study of five cases described palifermin-induced flexural hyperpigmentation — darkening of skin in body folds — with supporting biopsy findings (PMID 18795935). Broader drug reviews of palifermin in myelotoxic therapy-induced oral mucositis summarised the tolerability profile observed across the clinical programme (PMID 16225371), and a pharmacotherapy review covering its role in preventing chemotherapy- and radiation-induced mucositis discussed safety considerations alongside efficacy (PMID 17190850). Nursing literature noted that recognising these effects formed part of the supportive-care role when palifermin was used (PMID 17540295).

Quick Reference

Regulatory Context

Palifermin is a prescription biologic product, not a research-use-only compound. Reviews published after its approval described it in the context of myelotoxic therapy-induced oral mucositis in patients with haematological malignancies (PMID 16225371), and subsequent reviews discussed whether the evidence extended to other tumour types and treatment regimens (PMID 19607642). Administration in all published clinical work occurred under specialist oncology supervision.

Related Terms

• Keratinocyte growth factor (KGF-1 / FGF-7) — the naturally occurring parent protein.
• rHuKGF — the abbreviation used in pharmacology papers for the recombinant form (PMID 16765144).
• Oral mucositis — the epithelial injury endpoint measured in most palifermin trials (PMID 20694076).

This entry summarises published findings only and does not describe or endorse any use of palifermin. Readers with clinical questions should direct them to a licensed physician.

References

• Pharmacokinetic and pharmacodynamic interactions between palifermin and heparin (Journal of Clinical Pharmacology, 2015)
• Efficacy of palifermin (keratinocyte growth factor-1) in the amelioration of oral mucositis (Core Evidence, 2010)
• Palifermin-associated papular eruption (Archives of Dermatology, 2009)
• Palifermin in children undergoing autologous stem cell transplantation: a matched-pair analysis (Anticancer Research, 2014)
• Palifermin for patients with haematological malignancies: shifting nursing practice from symptom relief to prevention of oral mucositis (European Journal of Oncology Nursing, 2007)
• Pharmacokinetics, pharmacodynamics, and safety assessment of palifermin (rHuKGF) in healthy volunteers (Clinical Pharmacology and Therapeutics, 2006)
• Palifermin: a keratinocyte growth factor that reduces oral mucositis after stem cell transplant for haematological malignancies (Expert Opinion on Pharmacotherapy, 2006)
• Palifermin-induced flexural hyperpigmentation: a clinical and histological study of five cases (British Journal of Dermatology, 2008)
• Palifermin in the management of mucositis in hematological malignancies: current evidences and future perspectives (Cardiovascular & Hematological Agents in Medicinal Chemistry, 2009)
• Reduction of radiochemotherapy-induced early oral mucositis by recombinant human keratinocyte growth factor (palifermin): experimental studies in mice (International Journal of Radiation Oncology, Biology, Physics, 2005)
• Palifermin: in myelotoxic therapy-induced oral mucositis (Drugs, 2005)
• Palifermin: role in the prevention of chemotherapy- and radiation-induced mucositis (The Annals of Pharmacotherapy, 2007)