Kepivance (palifermin) / SOBI, Amgen - LARVOL DELTA

PREVENTION AND MANAGEMENT OF ORAL MUCOSITIS IN PEDIATRIC ONCO-IMMUNO-HEMATOLOGICAL PATIENTS: A SCOPING REVIEW (EBMT 2026) - "Other emerging treatments, including glutamine and palifermin, have demonstrated efficacy in reducing the incidence of mucositis... In conclusion, for the prevention and management of pediatric mucositis in the onco-immuno-hematological setting, there is a need to combine pharmacological and non-pharmacological treatments within a multidisciplinary care approach. Prevention consists of promoting oral hygiene educational interventions for young patients and caregivers through demonstrations and simulations. Education should be accompanied by targeted clinical oral examinations and the use of tailored products selected by dental hygienists, nurses, and physicians."

Clinical • Review • Bone Marrow Transplantation • Gene Therapies • Mucositis • Pediatrics • Stomatitis

PREVENTION AND MANAGEMENT OF ORAL MUCOSITIS IN PEDIATRIC ONCO-IMMUNO-HEMATOLOGICAL PATIENTS: A SCOPING REVIEW (EBMT 2026) - "Other emerging treatments, including glutamine and palifermin, have demonstrated efficacy in reducing the incidence of mucositis... In conclusion, for the prevention and management of pediatric mucositis in the onco-immuno-hematological setting, there is a need to combine pharmacological and non-pharmacological treatments within a multidisciplinary care approach. Prevention consists of promoting oral hygiene educational interventions for young patients and caregivers through demonstrations and simulations. Education should be accompanied by targeted clinical oral examinations and the use of tailored products selected by dental hygienists, nurses, and physicians."

Clinical • Review • Bone Marrow Transplantation • Gene Therapies • Mucositis • Pediatrics • Stomatitis

PREVENTION AND MANAGEMENT OF ORAL MUCOSITIS IN PEDIATRIC ONCO-IMMUNO-HEMATOLOGICAL PATIENTS: A SCOPING REVIEW (EBMT 2026) - "Other emerging treatments, including glutamine and palifermin, have demonstrated efficacy in reducing the incidence of mucositis... In conclusion, for the prevention and management of pediatric mucositis in the onco-immuno-hematological setting, there is a need to combine pharmacological and non-pharmacological treatments within a multidisciplinary care approach. Prevention consists of promoting oral hygiene educational interventions for young patients and caregivers through demonstrations and simulations. Education should be accompanied by targeted clinical oral examinations and the use of tailored products selected by dental hygienists, nurses, and physicians."

Clinical • Review • Bone Marrow Transplantation • Gene Therapies • Mucositis • Pediatrics • Stomatitis

APPROVED AND EMERGING OPTIONS FOR ONCOLOGY TREATMENT-RELATED TOXICITIES: A TARGETED LITERATURE REVIEW OF THE LANDSCAPE AND REMAINING UNMET NEEDS (ISPOR 2026) - "Agents were cataloged by intended use: (a) rescue therapies for acute overdose or early severe toxicity (e.g., glucarpidase, uridine triacetate, leucovorin rescue); (b) prophylactic organ-protective agents (e.g., mesna, amifostine, dexrazoxane, sodium thiosulfate, palifermin, trilaciclib); (c) countermeasures for immune-mediated toxicities (e.g., tocilizumab for cytokine release syndrome); and (d) radiation normal-tissue protectants in development (e.g., superoxide dismutase mimetics, avasopasem [GC-4419]). Across drug classes, agents demonstrated substantial clinical benefit in narrow, time-sensitive indications (e.g., rescue for high-dose methotrexate or fluoropyrimidine toxicity; cardioprotection with dexrazoxane; otoprotection with sodium thiosulfate in pediatric cisplatin-treated patients; and reduced mucositis with palifermin in select HSCT regimens)... Timely rescue administration can improve outcomes for patients experiencing severe treatment-related toxicities...."

Review • Bone Marrow Transplantation • Cardiovascular • Inflammation • Mucositis

Oral mucositis management in hematopoietic cell transplantation in Australia and New Zealand (CAN EAT Survey). (PubMed, Support Care Cancer) - "In Australia and New Zealand, national EviQ guidelines are used more often than the MASCC/ISOO guidelines. Feasibility for implementing palifermin and photobiomodulation should be reassessed, and greater collaboration in developing guidelines is necessary."

Journal • Mucositis • Pain • Stomatitis • Transplantation

Clinical evidence for managing radiotherapy-induced oral mucositis and xerostomia in head and neck cancer patients: a review of Phase III-IV trials. (PubMed, Int J Oral Maxillofac Surg) - "For oral mucositis, effective pharmacological agents included palifermin, avasopasem manganese, doxepin, morphine mouthwash, and gabapentin, while non-pharmacological options such as low-level laser therapy, honey, and Aloe vera showed additional benefit in reducing severity and improving healing. In managing radiotherapy-induced xerostomia, pilocarpine, amifostine, and cevimeline significantly enhanced salivary function, with adaptive radiotherapy techniques offering protective effects on the salivary glands...Although several interventions demonstrated promising clinical efficacy, the heterogeneity in study design, outcome measures, and follow-up duration limits definitive conclusions. Therefore, customized, evidence-driven approaches informed by existing recommendations and developed data are crucial for enhancing outcomes and reducing the burden of therapy-induced toxicities in head and neck cancer patients."

Journal • P3 data • Review • Dental Disorders • Head and Neck Cancer • Mucositis • Oncology • Oral Cancer • Solid Tumor • Stomatitis • Xerostomia

Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2 | N=82 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Dec 2026

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • Transplantation • CD34

Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant (clinicaltrials.gov) - P1 | N=53 | Recruiting | Sponsor: City of Hope Medical Center | Active, not recruiting ➔ Recruiting | Trial completion date: Oct 2025 ➔ Oct 2026 | Trial primary completion date: Oct 2025 ➔ Oct 2026

Enrollment open • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation • HLA-DRB1

Addition of Myeloablative Doses of Total Marrow Irradiation (TMI) to Myeloablative Fludarabine/Busulfan Conditioning for Allogeneic HSCT in Patients with High-Risk AML, CML, or Myelodysplastic Syndrome: A Phase II Clinical Trial (ASH 2023) - P2 | "The study included patients with matched related donors (MRD, n=9) receiving TMI/FluBu4 as a preparative regimen and patients with matched unrelated (MUD, n=13) or 1-antigen mismatched unrelated (MMUD, n=6) donors receiving conditioning with TMI/FluBu4 + rabbit anti-thymocyte globulin (rATG). Graft-versus-host disease (GVHD) prophylaxis was with methotrexate and tacrolimus, and palifermin was used to prevent severe mucositis...Kaplan Meyer curves for Overall Survival (OS) and DFS are shown in Figure 1 and 2, respectively. Addition of myeloablative TMI at 9Gy to standard myeloablative targeted FluBu4 was well tolerated and achieved very encouraging results in a group of patients with myeloid malignancies at high risk of relapse (clinicaltrials.gov Identifier: NCT03121014)."

Clinical • P2 data • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Mucositis • Myelodysplastic Syndrome • Oncology • Pneumonia • Respiratory Diseases • Septic Shock • Transplantation

Preconditioning Single High-Dose Palifermin Alters the Posttransplant Inflammatory Cytokine Profile (ASH 2023) - P1/2, P2 | "This trial evaluates the incidence of severe CGVHD (primary objective of phase 2) following the addition of a single high dose of palifermin to GVHD prophylaxis with TMS (tacrolimus, methotrexate, sirolimus)...All patients received cyclophosphamide (total 4.8 g/m2) and fludarabine (total 120 mg/m2) as conditioning on days −6 to −3 followed by infusion of an 8/8 HLA-matched unrelated donor peripheral blood cell graft on day 0...Further investigations focusing on GI microbiome and immune cell subsets are needed to elucidate the consequences of cytokine alterations induced by KGF on immune cell reconstitution. This research was supported by the Intramural Research Program of the NIH, NCI, CCR."

Post-transplantation • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Mucositis • Stomatitis • Transplantation • CD4 • CD8 • CXCL9 • IL12A • IL15 • IL22 • IL7 • TNFA

Comparison of Two Myeloablative Total-Body Irradiation (MAC-TBI) Regimens: 1200 Cgy Vs 800 Cgy of TBI for Allogeneic Stem Cell Transplantation in Adults with Hematological Malignancies (ASH 2024) - "The Fine-Gray model was used to correlate acute graft-versus-host disease (aGVHD) with clinical factors.Results : Twelve patients (52.2%) received fludarabine and TBI800, 9 patients (39.1%) received cyclophosphamide (Cy) and TBI1200, and 2 patients (8.7%) received fludarabine and TBI 1200 cGy. GVHD prophylaxis included : post-transplant Cy, mycophenolate mofetil, and tacrolimus in 5 patients (21.7%) and methotrexate and tacrolimus in 18 patients (78.3%). Palifermin was administered to 43.5% patients (25% in the TBI800 group vs 63.6% in the TBI1200 group, p=0.1)...The Multivariable Fine-Gray Model showed a higher risk of aGVHD, particularly grade I-II, in the TBI1200 group (HR 6.66, 95% CI : 2.07-21.38).Conclusion : TBI800 vs TBI1200 led to similar relapse incidence, with a lower risk of aGVHD and decreased grade 3-4 mucositis rate in the TBI800 group. Studies on a larger scale are required to establish the safety and efficacy of TBI800."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cataract • Dermatitis • Dermatology • Diabetes • Dyslipidemia • Febrile Neutropenia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Mucositis • Neutropenia • Novel Coronavirus Disease • Oncology • Ophthalmology • Osteoporosis • Respiratory Diseases • Rheumatology • Transplantation

Salivary gland tissue chip screening identifies candidate radioprotective drugs. (PubMed, Commun Med (Lond)) - "This body of work demonstrates the development and validation of assays using a tissue chip platform for high-content drug screening and the successful discovery and in vivo validation of candidate radioprotective drugs with non-antioxidant primary modes of action. These results point to possible unknown mechanisms of radioprotection. These drugs can be developed to improve radioprotection efficacy and clinical administration without adverse side-effects."

Journal • Dental Disorders • Head and Neck Cancer • Infectious Disease • Oncology • Solid Tumor • Xerostomia

Efficacy of Treatment and Prevention of Radiation-induced Oral Mucositis- Systematic Review (AAO-HNSF 2025) - "Review of these papers papers supports good oral care, IMRT, radiation shields, palifermin, and amifostine. To our knowledge, this study represents the largest review of RIOM. It is limited by the diversity of study designs, sample sizes, patient characteristics, and outcome measures. While there is no objectively universal treatment for RIOM, preliminary results from this review show promise with multiple therapies."

Clinical • Review • Head and Neck Cancer • Mucositis • Oncology • Stomatitis

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | Suspended ➔ Recruiting

Enrollment open • Immunology • Primary Immunodeficiency

Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study (clinicaltrials.gov) - P1/2 | N=1 | Recruiting | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | Enrolling by invitation ➔ Recruiting

Enrollment status • Gene Therapies • Immunology • Primary Immunodeficiency • CD40LG

Addressing Pain in Oral Mucositis: Narrative Review of Current Practices and Emerging Treatments. (PubMed, J Pain Res) - "Given the debilitating nature of OM in cancer patients and the critical need for effective pain control, this review aims to examine pharmacological advancements targeting the complex nature of OM-related pain, including agents such as lidocaine, doxepin, benzydamine, methylene blue, opioids, gabapentin, palifermin, caphosol, and ketamine...This review highlights the clinical applicability of emerging therapies, such as avasopasem manganese, which has demonstrated efficacy in mitigating OM progression...Addressing the multifaceted nature of OM-associated pain is essential for enhancing quality of life and optimizing cancer treatment outcomes. Further research is needed to establish robust, evidence-based guidelines for OM pain management."

Journal • Review • Bone Marrow Transplantation • Head and Neck Cancer • Infectious Disease • Mucositis • Oncology • Oral Cancer • Pain • Solid Tumor • Stomatitis • Transplantation

90Y-DOTA-anti-CD25 Basiliximab, Fludarabine, Melphalan, and Total Marrow and Lymphoid Irradiation for the Treatment of High-Risk Acute Leukemia or Myelodysplastic Syndrome (clinicaltrials.gov) - P1 | N=7 | Active, not recruiting | Sponsor: City of Hope Medical Center | Recruiting ➔ Active, not recruiting | N=12 ➔ 7

Enrollment change • Enrollment closed • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • FLT3 • IL2RA • ISG20 • NPM1

Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation (clinicaltrials.gov) - P1/2 | N=34 | Completed | Sponsor: National Cancer Institute (NCI) | Active, not recruiting ➔ Completed | Trial completion date: Dec 2027 ➔ Mar 2025

Trial completion • Trial completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • Transplantation • ABL1 • ASXL1 • BCR • CD34 • FLT3 • HLA-B • HLA-C

Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant (clinicaltrials.gov) - P1 | N=43 | Active, not recruiting | Sponsor: City of Hope Medical Center | Recruiting ➔ Active, not recruiting

Enrollment closed • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation • HLA-DRB1

Phase 1b/2a Trial of Allogeneic HSCT From an HLA-partially Matched Related or Unrelated Donor After TCRab+ T-cell/CD19+ B-cell Depletion for Patients With Monogenic and/or Early-onset Medically Refractory Crohn Disease (clinicaltrials.gov) - P1/2 | N=14 | Not yet recruiting | Sponsor: Stanford University

New P1/2 trial • Bone Marrow Transplantation • Crohn's disease • Gastroenterology • Genetic Disorders • Immunology • Inflammatory Bowel Disease • Transplantation

Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study (clinicaltrials.gov) - P1/2 | N=1 | Enrolling by invitation | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

New P1/2 trial • Gene Therapies • Immunology • Primary Immunodeficiency • CD40LG

Phase 1/2 Study of High-Dose Palifermin for GVHD Prophylaxis in Patients Undergoing HLA-Matched Unrelated Donor HCT. (PubMed, Blood) - P1/2 | "All 31 patients received fludarabine/cyclophosphamide RIC with tacrolimus, methotrexate, and sirolimus for GVHD prophylaxis. These findings indicate high-dose palifermin with RIC is safe and may prevent acute GVHD, though it did not impact chronic GVHD rates in this study. NCT02356159."

Journal • P1/2 data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation • CD4

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy (clinicaltrials.gov) - P1/2 | N=18 | Enrolling by invitation | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Gene Therapies • Genetic Disorders • Immunology • Primary Immunodeficiency

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | Trial completion date: Dec 2032 ➔ Dec 2034 | Trial primary completion date: Dec 2032 ➔ Dec 2034

Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Immunology • Primary Immunodeficiency

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (clinicaltrials.gov) - P1/2 | N=1 | Suspended | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | N=10 ➔ 1

Enrollment change • Immunology • Primary Immunodeficiency