TY - JOUR
T1 - Comparison of placebo and best available therapy for the treatment of myelofibrosis in the phase 3 COMFORT studies
AU - Mesa, Ruben A.
AU - Kiladjian, Jean Jacques
AU - Verstovsek, Srdan
AU - Al-Ali, Haifa Kathrin
AU - Gotlib, Jason
AU - Gisslinger, Heinz
AU - Levy, Richard
AU - Siulnik, Andres
AU - Gupta, Vikas
AU - Khan, Mahmudul
AU - DiPersio, John F.
AU - McQuitty, Mari
AU - Catalano, John V.
AU - Hunter, Deborah S.
AU - Knoops, Laurent
AU - Deininger, Michael
AU - Cervantes, Francisco
AU - Miller, Carole
AU - Vannucchi, Alessandro M.
AU - Silver, Richard T.
AU - Barbui, Tiziano
AU - Talpaz, Moshe
AU - Barosi, Giovanni
AU - Winton, Elliott F.
AU - Mendeson, Estella
AU - Harvey, Jimmie H.
AU - Arcasoy, Murat O.
AU - Hexner, Elizabeth
AU - Lyons, Roger M.
AU - Paquette, Ronald
AU - Raza, Azra
AU - Sun, William
AU - Sandor, Victor
AU - Kantarjian, Hagop M.
AU - Harrison, Claire
PY - 2014/2/1
Y1 - 2014/2/1
N2 - Prior to Janus kinase inhibitors, available therapies for myelofibrosis were generally supportive and did not improve survival. This analysis compares efficacy outcomes of patients with myelofibrosis in the control arms (placebo [n=154] and best available therapy [n=73]) from the two phase 3 COntrolled MyeloFibrosis study with ORal JAK inhibitor Treatment (COMFORT) studies. Spleen volume was assessed by magnetic resonance imaging/ computed tomography at baseline and every 12 weeks through week 72; spleen length was assessed by palpation at each study visit. Health-related quality of life and symptoms were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 Items at baseline and in weeks 4, 8, 12, 16 and 24 in COMFORT-I and in weeks 8, 16, 24 and 48 in COMFORT-II. The demographic and baseline characteristics were similar between the control arms of the two studies. One patient who received placebo and no patients who received best available therapy had a ≥35% reduction in spleen volume from baseline at week 24. At 24 weeks, neither placebo nor best available therapy had produced clinically meaningful changes in global quality of life or symptom scales. Non-hematologic adverse events were mostly grade 1/2; the most frequently reported adverse events in each group were abdominal pain, fatigue, peripheral edema and diarrhea. These data suggest that non-Janus kinase inhibitor therapies provide little improvement in splenomegaly, symptoms or quality of life as compared with placebo. Both COMFORT-I (NCT00952289) and COMFORT-II (NCT00934544) studies have been appropriately registered with clinicaltrials.gov.
AB - Prior to Janus kinase inhibitors, available therapies for myelofibrosis were generally supportive and did not improve survival. This analysis compares efficacy outcomes of patients with myelofibrosis in the control arms (placebo [n=154] and best available therapy [n=73]) from the two phase 3 COntrolled MyeloFibrosis study with ORal JAK inhibitor Treatment (COMFORT) studies. Spleen volume was assessed by magnetic resonance imaging/ computed tomography at baseline and every 12 weeks through week 72; spleen length was assessed by palpation at each study visit. Health-related quality of life and symptoms were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 Items at baseline and in weeks 4, 8, 12, 16 and 24 in COMFORT-I and in weeks 8, 16, 24 and 48 in COMFORT-II. The demographic and baseline characteristics were similar between the control arms of the two studies. One patient who received placebo and no patients who received best available therapy had a ≥35% reduction in spleen volume from baseline at week 24. At 24 weeks, neither placebo nor best available therapy had produced clinically meaningful changes in global quality of life or symptom scales. Non-hematologic adverse events were mostly grade 1/2; the most frequently reported adverse events in each group were abdominal pain, fatigue, peripheral edema and diarrhea. These data suggest that non-Janus kinase inhibitor therapies provide little improvement in splenomegaly, symptoms or quality of life as compared with placebo. Both COMFORT-I (NCT00952289) and COMFORT-II (NCT00934544) studies have been appropriately registered with clinicaltrials.gov.
UR - http://www.scopus.com/inward/record.url?scp=84896739915&partnerID=8YFLogxK
U2 - 10.3324/haematol.2013.087650
DO - 10.3324/haematol.2013.087650
M3 - Article
C2 - 23911705
AN - SCOPUS:84896739915
SN - 0390-6078
VL - 99
SP - 291
EP - 298
JO - Haematologica
JF - Haematologica
IS - 2
ER -