Patient goals, way to measure define CTCL success

September 1, 2004

New York - The key to successful management of cutaneous T-cell lymphoma (CTCL) is to identify the patient's goals, then find a way to measure response to treatment, Peter W. Heald, M.D., said here at the American Academy of Dermatology Academy '04 summer meeting.

New York - The key to successful management of cutaneous T-cell lymphoma (CTCL) is to identify the patient's goals, then find a way to measure response to treatment, Peter W. Heald, M.D., said here at the American Academy of Dermatology Academy '04 summer meeting.

"You have to define your goal, figure out a time course with whatever therapy you are working with, and then decide what parameter to follow to determine - yea or nay - it is working," says Dr. Heald, professor of dermatology, Yale University School of Medicine, New Haven, Conn.

Treatment goals in CTCL therapy fall into two different categories: palliation and remission; for each, the techniques of measuring success are different, Dr. Heald says.

Measuring the success of remittive therapy is "a little easier," Dr. Heald explains. "The main approach is to get rid of all lesions."

To assess the patient's response to therapy, investigators in clinical trials have typically quantified tumor burden using skin scoring techniques such as global mapping, severity recording or assessment of specific target lesions.

Palliation, in clinical trials of CTCL, is measured using questionnaires that are either general in nature or specific to the disease.

QOL measuresFor example, several quality-of-life measures were used in a recent trial of denileukin diftitox (ONTAK, Ligand Pharmaceuticals), including the Functional Assessment of Cancer Therapy-General (FACT-G) questionnaire, which measures overall quality of life; a seven-point skin appearance scale; and a 10 cm visual analogue scale of pruritus severity (Duvic M et al., Clin Lymphoma. 2002: 222).

Quality-of-life improvements were noted in the study, which included 71 patients with advanced or recurrent CTCL treated with an outpatient regimen of intravenous denileukin diftitox, given over 15 to 60 minutes for five consecutive days. The regimen was repeated every 21 days for a total of eight cycles.

In 21 documented responders, the composite FACT-G score gradually increased and reached statistical significance by the third cycle of treatment. Improvements were also noted in most FACT-G subscale scores (physical, social/family, emotional and functional well-being). Responders also had significant improvements in skin severity and pruritus scores.

The specific therapeutic course chosen - palliative versus remittive - will depend on the wishes of the patient and family, according to Dr. Heald.

"It's not preset," Dr. Heald tells Dermatology Times. "Any patient you see will have something to discuss regarding which way they want to go. Many factors play into that, including their age and their understanding of the disease."

In palliative therapy, the goal is to make the patient feel better, so a treatment such as interferon may not be appropriate due to its high side effect burden.

"Here, our goal is to try to keep this disease to a minimum, and we should have this achieved within two, four or six months," Dr. Heald says.

Topical treatment with bexarotene gel (Targretin, Ligand Pharmaceuticals) may not be appropriate for palliation, either, due to potential for skin irritation.