Prognostic factors and response to fludarabine therapy in patients with Waldenström macroglobulinemia: Results of United States intergroup trial (Southwest Oncology Group S9003)

Madhav V. Dhodapkar, Joth L. Jacobson, Morie A. Gertz, Saul E. Rivkin, G. David Roodman, Joseph M. Tuscano, Muhammad Shurafa, Robert A. Kyle, John J. Crowley, Bart Barlogie

Research output: Contribution to journalArticle

121 Citations (Scopus)

Abstract

Current information on Waldenström macroglobulinemia (WM) is based on retrospective or single-institution studies of patients requiring therapy. Between 1992 and 1998, 231 patients with WM were enrolled in a prospective observational multicenter clinical trial. Of these, 182 patients with symptomatic or progressive disease were treated with 4 to 8 cycles of therapy with a purine nucleoside analogue, fludarabine (FAMP; 30 mg/m 2 Of body-surface area daily for 5 days every 28 days). A serum β2-microglobulin (β2M) level below 3 mg/L and a hemoglobin level of at least 120 g/L (12 g/dL) at presentation predicted a lower likelihood of requiring therapy. The overall rate of response to FAMP therapy was 36% (95% confidence interval, 29%-44%), with 2% complete remissions. Patients who were 70 years old or older had a substantially lower likelihood of response (odds ratio, 0.34; P .004) than younger patients. On multivariate analysis, a serum β2M level of 3 mg/L or higher, hemoglobin level below 120 g/L, and serum IgM level below 40 g/L [4 g/dL] were significant adverse prognostic factors for survival. We developed a simple staging system for WM by using these variables and identified 4 distinct subsets of patients with estimated 5-year overall survival rates of 87%, 64%, 53%, and 22%, and 5-year progression-free survival rates of 83%, 55%, 33%, and 12%. Prognosis in WM is highly variable and serum β2M was the dominant predictor of a need for therapy and of survival. FAMP has activity against WM. Our staging system may provide guidance for a risk-based approach to the treatment of WM.

Original languageEnglish (US)
Pages (from-to)41-48
Number of pages8
JournalBlood
Volume98
Issue number1
DOIs
StatePublished - Jul 1 2001
Externally publishedYes

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Waldenstrom Macroglobulinemia
Oncology
Hemoglobins
Purine Nucleosides
Immunoglobulin M
Serum
Therapeutics
Survival Rate
Survival
Body Surface Area
Multicenter Studies
Disease-Free Survival
fludarabine
Multivariate Analysis
Odds Ratio
Clinical Trials
Confidence Intervals

ASJC Scopus subject areas

  • Hematology

Cite this

Prognostic factors and response to fludarabine therapy in patients with Waldenström macroglobulinemia : Results of United States intergroup trial (Southwest Oncology Group S9003). / Dhodapkar, Madhav V.; Jacobson, Joth L.; Gertz, Morie A.; Rivkin, Saul E.; Roodman, G. David; Tuscano, Joseph M.; Shurafa, Muhammad; Kyle, Robert A.; Crowley, John J.; Barlogie, Bart.

In: Blood, Vol. 98, No. 1, 01.07.2001, p. 41-48.

Research output: Contribution to journalArticle

Dhodapkar, Madhav V. ; Jacobson, Joth L. ; Gertz, Morie A. ; Rivkin, Saul E. ; Roodman, G. David ; Tuscano, Joseph M. ; Shurafa, Muhammad ; Kyle, Robert A. ; Crowley, John J. ; Barlogie, Bart. / Prognostic factors and response to fludarabine therapy in patients with Waldenström macroglobulinemia : Results of United States intergroup trial (Southwest Oncology Group S9003). In: Blood. 2001 ; Vol. 98, No. 1. pp. 41-48.
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abstract = "Current information on Waldenstr{\"o}m macroglobulinemia (WM) is based on retrospective or single-institution studies of patients requiring therapy. Between 1992 and 1998, 231 patients with WM were enrolled in a prospective observational multicenter clinical trial. Of these, 182 patients with symptomatic or progressive disease were treated with 4 to 8 cycles of therapy with a purine nucleoside analogue, fludarabine (FAMP; 30 mg/m 2 Of body-surface area daily for 5 days every 28 days). A serum β2-microglobulin (β2M) level below 3 mg/L and a hemoglobin level of at least 120 g/L (12 g/dL) at presentation predicted a lower likelihood of requiring therapy. The overall rate of response to FAMP therapy was 36{\%} (95{\%} confidence interval, 29{\%}-44{\%}), with 2{\%} complete remissions. Patients who were 70 years old or older had a substantially lower likelihood of response (odds ratio, 0.34; P .004) than younger patients. On multivariate analysis, a serum β2M level of 3 mg/L or higher, hemoglobin level below 120 g/L, and serum IgM level below 40 g/L [4 g/dL] were significant adverse prognostic factors for survival. We developed a simple staging system for WM by using these variables and identified 4 distinct subsets of patients with estimated 5-year overall survival rates of 87{\%}, 64{\%}, 53{\%}, and 22{\%}, and 5-year progression-free survival rates of 83{\%}, 55{\%}, 33{\%}, and 12{\%}. Prognosis in WM is highly variable and serum β2M was the dominant predictor of a need for therapy and of survival. FAMP has activity against WM. Our staging system may provide guidance for a risk-based approach to the treatment of WM.",
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AU - Jacobson, Joth L.

AU - Gertz, Morie A.

AU - Rivkin, Saul E.

AU - Roodman, G. David

AU - Tuscano, Joseph M.

AU - Shurafa, Muhammad

AU - Kyle, Robert A.

AU - Crowley, John J.

AU - Barlogie, Bart

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N2 - Current information on Waldenström macroglobulinemia (WM) is based on retrospective or single-institution studies of patients requiring therapy. Between 1992 and 1998, 231 patients with WM were enrolled in a prospective observational multicenter clinical trial. Of these, 182 patients with symptomatic or progressive disease were treated with 4 to 8 cycles of therapy with a purine nucleoside analogue, fludarabine (FAMP; 30 mg/m 2 Of body-surface area daily for 5 days every 28 days). A serum β2-microglobulin (β2M) level below 3 mg/L and a hemoglobin level of at least 120 g/L (12 g/dL) at presentation predicted a lower likelihood of requiring therapy. The overall rate of response to FAMP therapy was 36% (95% confidence interval, 29%-44%), with 2% complete remissions. Patients who were 70 years old or older had a substantially lower likelihood of response (odds ratio, 0.34; P .004) than younger patients. On multivariate analysis, a serum β2M level of 3 mg/L or higher, hemoglobin level below 120 g/L, and serum IgM level below 40 g/L [4 g/dL] were significant adverse prognostic factors for survival. We developed a simple staging system for WM by using these variables and identified 4 distinct subsets of patients with estimated 5-year overall survival rates of 87%, 64%, 53%, and 22%, and 5-year progression-free survival rates of 83%, 55%, 33%, and 12%. Prognosis in WM is highly variable and serum β2M was the dominant predictor of a need for therapy and of survival. FAMP has activity against WM. Our staging system may provide guidance for a risk-based approach to the treatment of WM.

AB - Current information on Waldenström macroglobulinemia (WM) is based on retrospective or single-institution studies of patients requiring therapy. Between 1992 and 1998, 231 patients with WM were enrolled in a prospective observational multicenter clinical trial. Of these, 182 patients with symptomatic or progressive disease were treated with 4 to 8 cycles of therapy with a purine nucleoside analogue, fludarabine (FAMP; 30 mg/m 2 Of body-surface area daily for 5 days every 28 days). A serum β2-microglobulin (β2M) level below 3 mg/L and a hemoglobin level of at least 120 g/L (12 g/dL) at presentation predicted a lower likelihood of requiring therapy. The overall rate of response to FAMP therapy was 36% (95% confidence interval, 29%-44%), with 2% complete remissions. Patients who were 70 years old or older had a substantially lower likelihood of response (odds ratio, 0.34; P .004) than younger patients. On multivariate analysis, a serum β2M level of 3 mg/L or higher, hemoglobin level below 120 g/L, and serum IgM level below 40 g/L [4 g/dL] were significant adverse prognostic factors for survival. We developed a simple staging system for WM by using these variables and identified 4 distinct subsets of patients with estimated 5-year overall survival rates of 87%, 64%, 53%, and 22%, and 5-year progression-free survival rates of 83%, 55%, 33%, and 12%. Prognosis in WM is highly variable and serum β2M was the dominant predictor of a need for therapy and of survival. FAMP has activity against WM. Our staging system may provide guidance for a risk-based approach to the treatment of WM.

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