Langner, S., Staber, P.B., Schub, N., Gramatzki, M., Grothe, W., Behre, G., … Neumeister, P. (2008). Palifermin reduces incidence and severity of oral mucositis in allogeneic stem-cell transplant recipients. Bone Marrow Transplantation, 42, 275–279.

DOI Link

Intervention Characteristics/Basic Study Process

Palifermin was administered at 60 mcg/kg per day for three consecutive days before the initiation of conditioning therapy and again after graft infusion.

Sample Characteristics

A group of 30 patients who had been treated with palifermin and undergone allogeneic hematopoietic stem-cell transplantation (HSCT) were retrospectively compared to a control group of 30 consecutive untreated patients receiving myeloablative conditioning.

  • Median age of patients was 38 years old, with a range was 18–59 years.
  • Patients had the following diagnoses.
    • Acute myelogenous leukemia (AML)/myelodysplastic syndrome (MDS) (n = 40)
    • Acute lymphocytic leukemia (ALL) (n = 14)
    • Chronic myelogenous leukemia (CML) (n = 5)
    • Chronic myelomonocytic (CMML) (n = 1)
  • Patients received allogeneic HSCT after CY/TBI or chemotherapy solely.
  • No growth factors were given.

Setting

The study was conducted from May 2005 to December 2006 in Austria and Germany.

Study Design

The study was conducted within a compassionate use program.

Measurement Instruments/Methods

The World Health Organization (WHO) Oral Toxicity Scale was used.

Results

  • Incidence of grade 2–4 WHO oral toxicity was 60% in the palifermin group and 86% in the control group (p = 0.04).
  • Grade 3–4 oral toxicity was present in 37% of the palifermin group and 53% of the control group (p = 0.19).
  • Mean duration of oral mucositis (OM) was 6 versus 12 days (p = 0.003) in favor of palifermin. 
  • Severity of OM was significantly reduced in the study group at 1.73 versus 2.47 (p = 0.03).
  • Palifermin was associated with significantly decreased use of opioids. The median cumulative dose  was 150 versus 378 morphine equivalents (p = 0.04). However, the difference in median time of use was not significant at 6 versus 7 (p = NS).
  • Total parenteral nutrition (TPN) use was lower in the palifermin group at 26 versus 15 days (p = 0.002). 
  • No significant difference was found in incidence and duration of febrile neutropenia, with the palifermin group at 4 days versus 3 days in the control group (p = NS).
  • Hematopoietic recovery was not influenced.
  • No significant difference was found in graft-versus-host disease (GVHD).

Limitations

  • The authors declared no funding or financial support by company.
  • An historical control group was used.
  • The sample size was small.
  • No discussion of added costs was included.