Title

Using Theophylline to Treat Patients With Early Stage 0 or IB Chronic Lymphocytic Leukemia (A Phase II Trial)

Sponsor

Eastern Cooperative Oncology Group through the NCI-sponsored Cancer Cooperative Group Program

Purpose of the Study

The purpose of this study was to evaluate the effectiveness of theophylline, a drug commonly used to treat asthma, as a therapy for patients with early stage chronic lymphocytic leukemia and to make sure the side effects are not too severe.

Results

The study was stopped after 25 patients were treated because by that time no patient had a complete response, meaning their disease was no longer detected, or partial response, meaning disease got better but was still detectable. One patient had a complete response after the study was over. The disease stayed the same (did not progress) in 72 percent of study participants and got worse in 24 percent. Progression-free survival was 17.8 months (the time at which the cancer had gotten worse in half the participants and remained stable in the other half). Another finding in the study was that patients whose cancer cells were able to be destroyed by theophylline in the laboratory had a better chance that their cancer would respond to this treatment and remain stable.

Conclusion

Although interesting, theophylline will not be developed further at this time primarily because there currently are many newer, more effective agents, such as Rituxan, Campath, and lenolidomide, for CLL that have a higher priority.

Start Date

May 1999

Stop Date

June 2000

Number of Participating Patients

25

Eligibility Requirements

Participants in this study were at least 18 years old and had no previous chemotherapy.

Study Design

Patients took theophylline pills (200 mg.) every twelve hours for as long as their cancer responded to treatment (got better) or remained the same. Patients were evaluated every four weeks. Patients whose disease got worse were taken off the study, but continued follow-up.

Side Effects

Nineteen patients had only mild or moderate side effects. Four patients had severe side effects. One patient had headache and one patient had loss of appetite, nausea, and vomiting. Two patients had elevated liver function tests and neutrophils. No life-threatening side effects occurred.

References

Wiernik P, Paietta EM, Goloubeva O, Lee S, Makower D, Bennett JM, Wade JL, Ghosh C, Kaminer LS, Pizzolo J, Tallman MS. Phase II Study of Theophylline in Chronic Lymphocytic Leukemia: A Study of the Eastern Cooperative Oncology Group (E4998). Leukemia 2004 Oct;18(10):1605-10. (E4998) [Manuscript published 10/15/2004]

National Cancer Institute Patient Summary

http://www.cancer.gov/clinical_trials/view_clinicaltrials.aspx?version=patient&cdrid=66953

Reviewed by

Peter Wiernik, MD, Our Lady of Mercy Medical Center, Bronx, NY; Robert Gray, PhD, ECOG Statistician; Mary Lou Smith, ECOG Patient Representative

Released May 29, 2006 (Revised July 19, 2006)