Kurzrock, R., Cortes, J., Thomas, D.A., Jeha, S., Pilat, S., & Talpaz, M. (2001). Pilot study of low-dose interleukin-11 in patients with bone marrow failure. Journal of Clinical Oncology, 19, 4165–4172.

Intervention Characteristics/Basic Study Process

  • Interleukin-11 (IL-11) 10 mcg/kg subcutaneous was given daily for two weeks followed by a two-week rest period, then dosing was repeated. After the first two courses (eight weeks), patients who responded continued maintenance therapy with which length of course, rest period, and doses were individualized based on response and side effects. Maintain platelet 150–450.
  • Platelet count equation = x 109 /L
  • During therapy, CBC with differential and reticulocyte count was done three times per week for the first six weeks and then at least weekly.
  • Patients were not transfused if platelets were greater than 10.
  • Patient responses had to last at least four weeks while on therapy.
  • Median baseline platelet = 12
     

Sample Characteristics

  • N = 16
  • AGE = 5–84 years 
  • MALES: 14, FEMALES: 4
  • Two patients were registered in error.
  • OTHER KEY SAMPLE CHARACTERISTICS: Patients with myelodysplastic syndromes (MDS), aplastic anemia, graft failure, postchemotherapy aplasia, refractory anemia, bone marrow failure after autologous bone marrow transplant. No chemotherapy for two months or disease progression (other than worsening MDS) with platelets less than or equal to 50



 

Measurement Instruments/Methods

  • Raise platelet count without significant toxicity.
  • Response was doubling of platelets if baseline was 20–50 or tripling if baseline was less than or equal to 20.
  • Baseline = median of three untransfused counts within two weeks before starting therapy
     

Results

Six of 16 patients (38%) showed an increase in platelet count greater than 70K, mostly within the first two-week course. Of the six responders, their baseline platelet count was 1–48; none received transfusions. One patient also received granulocyte colony-stimulating factor and erythropoietin; time to response was approximately 20 weeks. Two patients had an abrupt decrease in platelet counts in the two-week rest period between courses. Response duration was 12–30 weeks. Side effects were mild peripheral edema (two patients treated with furosemide 20 mg PO), conjunctival infection, and myalgia; seven patients had no side effects.
 

Limitations

  • Small study size (16)
  • Six case studies' responders presented
  • Subset of patients with bone marrow failure, mild thrombocytopenia
  • Pilot study with review of existing literature
  • No control
  • Not randomized