TY - JOUR
T1 - Use of the functional assessment of cancer therapy-anemia in persons with myeloproliferative neoplasm-associated myelofibrosis and anemia
AU - Tefferi, Ayalew
AU - Hudgens, Stacie
AU - Mesa, Ruben
AU - Peter Gale, Robert
AU - Verstovsek, Srdan
AU - Passamonti, Francesco
AU - Cervantes, Francisco
AU - Rivera, Candido
AU - Tencer, Tom
AU - Khan, Zeba M.
N1 - Funding Information:
Drs. Tefferi, Passamonti, Cervantes, and Rivera have not conflicts of interest in regards to the content of this article. Ms. Hudgens is employed by Adelphi Values (Healthcare Consultancy). Dr. Mesa received research support from Celgene, Incyte, Lilly, Genentech, NS Pharma, and Gilead. Dr. Gale is a part-time employee of Celgene. Dr. Verstovsek received research support from Celgene for conduct of clinical studies. Drs. Tencer and Khan are employed by Celgene. Research Study and preparation of typescript was funded by Celgene Corporation.
PY - 2014/4/1
Y1 - 2014/4/1
N2 - Background Anemia is common in myeloproliferative neoplasm (MPN)-associated myelofibrosis. The Functional Assessment of Cancer Therapy (FACT) measurement system is a patient-reported outcomes instrument that documents symptoms of the diverse aspects of cancer treatment. One FACT version, FACT-Anemia (FACT-An), documents symptoms of anemia related to cancer. The FACT-An has been validated in diverse cancer populations, but not in MPN-associated myelofibrosis. Objective Our aim was to evaluate the relationship between anemia response to therapy with pomalidomide with or without corticosteroids and patient-reported outcomes using the FACT-An instrument. Methods Data were obtained from a Phase II, randomized, double-blind Bayesian pick-the-winner trial of prednisone and pomalidomide in patients with MPN-associated myelofibrosis and anemia (red blood cell-transfusion dependence). Details of the study, including definitions of anemia, anemia response, red blood cell-transfusion, red blood cell-transfusion dependence, and red blood cell-transfusion independence, are reported. Change in quality of life from randomization to the last cycle of therapy was evaluated using the FACT-An Physical Well Being, Functional Well Being, Trial Outcome Index, and Anemia domains. Clinically important differences were used to determine the smallest difference in scores that patients perceived as beneficial in the FACT-An domains of interest. Patients were classified as meeting clinically important differences for responsiveness if their change score from baseline was >1 SEM, indicating improvement. Results Eighty-five patients were studied. Thirty-one patients (37%) were classified as anemia responders by prospectively defined criteria. Across all FACT-An domains, anemia responders showed greater improvement in Physical Well Being, Functional Well Being, and Trial Outcome Index scores than did nonresponders. This improvement began at the second 28-day cycle of therapy and was sustained. Conclusions We show a correlation between anemia response and improved quality of life measured by the FACT-An instrument in patients with MPN-associated myelofibrosis and anemia.
AB - Background Anemia is common in myeloproliferative neoplasm (MPN)-associated myelofibrosis. The Functional Assessment of Cancer Therapy (FACT) measurement system is a patient-reported outcomes instrument that documents symptoms of the diverse aspects of cancer treatment. One FACT version, FACT-Anemia (FACT-An), documents symptoms of anemia related to cancer. The FACT-An has been validated in diverse cancer populations, but not in MPN-associated myelofibrosis. Objective Our aim was to evaluate the relationship between anemia response to therapy with pomalidomide with or without corticosteroids and patient-reported outcomes using the FACT-An instrument. Methods Data were obtained from a Phase II, randomized, double-blind Bayesian pick-the-winner trial of prednisone and pomalidomide in patients with MPN-associated myelofibrosis and anemia (red blood cell-transfusion dependence). Details of the study, including definitions of anemia, anemia response, red blood cell-transfusion, red blood cell-transfusion dependence, and red blood cell-transfusion independence, are reported. Change in quality of life from randomization to the last cycle of therapy was evaluated using the FACT-An Physical Well Being, Functional Well Being, Trial Outcome Index, and Anemia domains. Clinically important differences were used to determine the smallest difference in scores that patients perceived as beneficial in the FACT-An domains of interest. Patients were classified as meeting clinically important differences for responsiveness if their change score from baseline was >1 SEM, indicating improvement. Results Eighty-five patients were studied. Thirty-one patients (37%) were classified as anemia responders by prospectively defined criteria. Across all FACT-An domains, anemia responders showed greater improvement in Physical Well Being, Functional Well Being, and Trial Outcome Index scores than did nonresponders. This improvement began at the second 28-day cycle of therapy and was sustained. Conclusions We show a correlation between anemia response and improved quality of life measured by the FACT-An instrument in patients with MPN-associated myelofibrosis and anemia.
KW - FACT-An
KW - MPN-associated myelofibrosis
KW - anemia
KW - cancer
KW - clinical trial
KW - fatigue
KW - patient-reported outcome
KW - prednisone
KW - quality of life
KW - responsiveness
KW - validation study
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U2 - 10.1016/j.clinthera.2014.02.016
DO - 10.1016/j.clinthera.2014.02.016
M3 - Article
C2 - 24636526
AN - SCOPUS:84898918548
SN - 0149-2918
VL - 36
SP - 560
EP - 566
JO - Clinical therapeutics
JF - Clinical therapeutics
IS - 4
ER -