Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: PEG-Intron For Chronic Myelogenous Leukemia Patients Unresponsive To Or Intolerant Of Roferon Or Intron

Summary: The purpose of this study is to determine if PEG-Intron is better tolerated and more efficacious than standard interferons (Roferon, Intron) in patients with Philadelphia-positive Chronic Myelogenous Leukemia. These patients should have previously received standard interferon therapy and have been intolerant, resistant, or have relapsed disease.

Detailed description: It has been shown that patients who experience complete hematologic or at least a partial cytogenetic response to interferon will have improved survival times. In addition, evidence exists that even patients who do not demonstrate a cytogenetic response to interferon treatment can still benefit from treatment, in terms of survival, compared to patients not treated with interferon. This indicates that if a patient is better able to tolerate interferon, he or she may have improved survival even without cytogenetic response. Preliminary studies suggest that PEG-Intron is more convenient for patients (administered once weekly rather than daily), is better tolerated than interferon, and can produce hematologic remission in interferon-a resistant patients. Phase II studies are needed to ascertain the overall hematologic and cytogenetic response rates to PEG-Intron in such patients.

Eligibility: Inclusion Criteria:
  -  Chronic phase CML, documented by the presence of Philadelphia chromosome or bcr/abl rearrangement at time of diagnosis, confirmed by either cytogenetics or PCR.
  -  WBC >/= 3000/ul </=100,000/ul.
  -  Patients must have received prior interferon therapy & proven to have primary refractory disease, secondary resistance or intolerance to interferon-a
  -  Patient must have ECOG status of 0, 1, or 2
  -  Labs: SGOT/SGPT<2xULN; serum bilirubin<2xULN; serum creatinine <2.0mg/dl
  -  Recovered from effects of major surgery
  -  Life expectancy > 12 wks.
  -  Signed informed consent.
  -  Women of childbearing potential must have negative serum pregnancy test within 72 hrs prior to administration of PEG-Intron & use effective contraception during the study.
  Exclusion Criteria:
  -  NO accelerated Phase CML patients with peripheral blood: blasts>/=15%, basophils>/=20%, blasts+promyelocytes>/=30%, platelets<100,000/ul (unrelated to therapy). Blastic phase CML:>/=30% in peripheral blood/bone marrow.
  -  NO patients with known hypersensitivity to interferon-a.
  -  NO severe cardiovascular disease, i.e. arrhythmias requiring chronic treatment or congestive heart failure (NYHA classification III/IV).
  -  NO history of neuropsychiatric disorder requiring hospitalization.
  -  NO patients requiring therapy for refractory thyroid dysfunction
  -  NO patients with uncontrolled diabetes mellitus.
  -  NO patients who have had treatment for a 2nd malignancy in the past 5 yrs, except for localized basal cell/squamous cell carcinoma of the skin or cervical carcinoma in situ.
  -  NO pregnant or lactating patients.
  -  NO patients known to be actively using alcohol or drugs
  -  NO patients receiving any experimental therapy within 30 days of enrollment in study.

Relevance to patient case: possibly relevant