Profile of pomalidomide and its potential in the treatment of myelofibrosis

Figures & data

Table 1 Comparison of response rates amongst completed clinical trials for pomalidomide in myelofibrosis

Reference	Phase	Dosing regimen and sample size	Sample size (n)	Criteria	Anemia improvement (%)	Splenomegaly improvement (%)
Tefferi et al^Citation53	II	Pomalidomide 0.5 mg + prednisone	22	IWG-MRT	36	0
		Pomalidomide 2 mg alone	22	IWG-MRT	23	0
		Prednisone alone	21	IWG-MRT	19	0
		Pomalidomide 2 mg + prednisone	19	IWG-MRT	16	0
Mesa et al^Citation54	I/II	Pomalidomide 0.5 mg	2	IWG-MRT	63	29
		Pomalidomide ≥2.5 mg	11	IWG-MRT	18	22
Begna et al^Citation55	II	Pomalidomide 0.5 mg	58	IWG-MRT	17	0
Daver et al^Citation57	II	Pomalidomide 3.0 mg	21	IWG-MRT CI	NR	NR
		Pomalidomide 0.5 mg	29	Delphi	10	0
Daver et al^Citation58	II	Pomalidomide 0.5 mg + prednisone	29	Delphi	22	0

Abbreviations: CI, clinical improvement; IWG-MRT, International Working Group for Myelofibrosis Research and Treatment; NR, not reported.

Table 2 Comparison of immunomodulatory agents

IMiD	Phase	Sample size (n)	Dose	Adverse effects	Anemia response rate	Spleen response
Thalidomide^Citation34 (with prednisone)	II	21	50 mg/day	Constipation (38%) Somnolence (29%) Grade 2 neuropathy (29%)	62%	19%
Lenalidomide^Citation50 (with prednisone)	II	40	10 mg/day	Grade ≥3: Neutropenia (58%) Anemia (42%) Fatigue (27%) Thrombocytopenia (13%)	30%	42%
Pomalidomide^Citation53 (with prednisone)	II	22	0.5 mg/day	Grade ≥3: Neutropenia (8%) Thrombocytopenia (11%) Pneumonia/sepsis (11%) Venous thromboembolism (4%)	36%	0

Abbreviation: IMiD, immunomodulatory medication.

TefferiAVerstovsekSBarosiGPomalidomide is active in the treatment of anemia associated with myelofibrosisJ Clin Oncol200927274563456919652059

 PubMed Web of Science ®Google Scholar

MesaRAPardananiADHusseinKPhase 1/-2 study of pomalidomide in myelofibrosisAm J Hematol201085212913020052748

 PubMed Web of Science ®Google Scholar

BegnaKHMesaRAPardananiAA phase-2 trial of low-dose pomalidomide in myelofibrosisLeukemia201125230130421052089

 PubMed Web of Science ®Google Scholar

DaverNShastriAKadiaTModest activity of pomalidomide in patients with myelofibrosis and significant anemiaLeuk Res201337111440144423890523

 PubMed Web of Science ®Google Scholar

DaverNShastriAKadiaTPhase II study of pomalidomide in combination with prednisone in patients with myelofibrosis and significant anemiaLeuk Res20143891126112925047979

 PubMed Web of Science ®Google Scholar

MesaRASteensmaDPPardananiAA phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasiaBlood200310172534254112517815

 PubMed Web of Science ®Google Scholar

Quintas-CardamaAKantarjianHMManshouriTLenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosisJ Clin Oncol200927284760476619720904

 PubMed Web of Science ®Google Scholar