title: Safe and prolonged survival with long-term exposure to pomalidomide in relapsed/refractory myeloma.
abstract: The IFM2009-02 trial studied pomalidomide (4 mg daily, 21/28 versus 28/28) and dexamethasone in very advanced relapsed or refractory multiple myeloma (RRMM). We observed that 40% of patients had a prolonged progression-free survival (PFS) and subsequently overall survival (OS). We sought to analyze the characteristics of these patients and study the effect of long exposure to pomalidomide.
  
  
  
  We separated the studied population into two groups: 3 months to 1 year (<1 year) and more than 1 year (≥1 year) of treatment with pomalidomide and dexamethasone based on clinical judgment and historical control studies. We then analyzed the characteristics of patients according to duration of treatment.
  
  
  
  The overall response rate (ORR) for the <1-year group was 43%, the median PFS 4.6 months [95% confidence interval (95% CI) 3.8-6.4] with only 6% at 12 months, and the median OS was 15 months (11.7-20.3) and 40% at 18 months. For the ≥1-year group, the response rate and survival were strikingly different, ORR at 83%, median PFS 20.7 months (14.7-35.4), median OS not reached, and 91% at 18 months.
  
  
  
  Pomalidomide and dexamethasone favored prolonged and safe exposure to treatment in 40% of heavily treated and end-stage RRMM, a paradigm shift in the natural history of RRMM characterized with a succession of shorter disease-free intervals and ultimately shorter survival. Although an optimization of pomalidomide-dexamethasone regimen is warranted in advanced RRMM, we claim that pomalidomide has proven once more to change the natural history of myeloma in this series, which should be confirmed in a larger study.
authors.affiliation: Department of Blood Diseases, Hôpital Huriez, CHRU Lille, Lille.
authors.forename: G
authors.initials: G
authors.lastname: Fouquet
authors.affiliation: Department of Hematology, Hôpital A.Michallon, CHU Grenoble, Grenoble.
authors.forename: B
authors.initials: B
authors.lastname: Pegourie
authors.affiliation: Department of Hematology, Hôpital Côte de Nacre, CHU Caen, Caen.
authors.forename: M
authors.initials: M
authors.lastname: Macro
authors.affiliation: Department of Blood Diseases, Hôpital Huriez, CHRU Lille, Lille.
authors.forename: M O
authors.initials: MO
authors.lastname: Petillon
authors.affiliation: Department of Hematology, Centre Hospitalier Lyon-Sud, Lyon.
authors.forename: L
authors.initials: L
authors.lastname: Karlin
authors.affiliation: Department of Hematology, CHU de Dijon, Dijon.
authors.forename: D
authors.initials: D
authors.lastname: Caillot
authors.affiliation: Department of Hematology, CHU de Toulouse, Toulouse.
authors.forename: M
authors.initials: M
authors.lastname: Roussel
authors.affiliation: Department of Hematology, Hôpital Saint Louis, APHP, Paris.
authors.forename: B
authors.initials: B
authors.lastname: Arnulf
authors.affiliation: Intergroupe Francophone du Myélome (IFM).
authors.forename: C
authors.initials: C
authors.lastname: Mathiot
authors.affiliation: Department of Hematology and Cell Therapy Service, CHU de Bordeaux, Pessac, France.
authors.forename: G
authors.initials: G
authors.lastname: Marit
authors.affiliation: Department of Hematology, Hôpital Robert Debré, CHU Reims, Reims.
authors.forename: B
authors.initials: B
authors.lastname: Kolb
authors.affiliation: Department of Hematology, Institut Paoli Calmettes, Marseille.
authors.forename: A M
authors.initials: AM
authors.lastname: Stoppa
authors.affiliation: Department of Hematology, CHU Avicennes, APHP, Paris.
authors.forename: S
authors.initials: S
authors.lastname: Brechiniac
authors.affiliation: Department of Hematology, CHU Nice, Nice.
authors.forename: V
authors.initials: V
authors.lastname: Richez
authors.affiliation: Department of Hematology, CH de Périgueux, Périgueux.
authors.forename: P
authors.initials: P
authors.lastname: Rodon
authors.affiliation: Department of Hematology, CH de la Côte Basque, Bayonne.
authors.forename: A
authors.initials: A
authors.lastname: Banos
authors.affiliation: Department of Hematology, CH de Dunkerque, Dunkerque.
authors.forename: M
authors.initials: M
authors.lastname: Wetterwald
authors.affiliation: Department of Hematology, CHU St-Antoine, Paris.
authors.forename: L
authors.initials: L
authors.lastname: Garderet
authors.affiliation: Department of Hematology, Hôpital Sud, CHU Amiens, Amiens.
authors.forename: B
authors.initials: B
authors.lastname: Royer
authors.affiliation: Department of Hematology, Hôpital de Nancy, Nancy.
authors.forename: C
authors.initials: C
authors.lastname: Hulin
authors.affiliation: Department of Hematology, Hôpital de Tours, Tours.
authors.forename: L
authors.initials: L
authors.lastname: Benbouker
authors.affiliation: Department of Hematology, CHU Rennes, Rennes.
authors.forename: O
authors.initials: O
authors.lastname: Decaux
authors.affiliation: Department of Hematology, CHU Rennes, Rennes.
authors.forename: M
authors.initials: M
authors.lastname: Escoffre-Barbe
authors.affiliation: Department of Hematology, Hôpital Saint Louis, APHP, Paris.
authors.forename: J P
authors.initials: JP
authors.lastname: Fermand
authors.affiliation: Department of Hematology, CHU de Toulouse, Toulouse.
authors.forename: M
authors.initials: M
authors.lastname: Attal
authors.affiliation: Department of Hematology, CHU de Toulouse, Toulouse.
authors.forename: H
authors.initials: H
authors.lastname: Avet-Loiseau
authors.affiliation: Department of Hematology, CHU Nantes, Nantes.
authors.forename: P
authors.initials: P
authors.lastname: Moreau
authors.affiliation: Department of Blood Diseases, Hôpital Huriez, CHRU Lille, Lille.
authors.forename: T
authors.initials: T
authors.lastname: Facon
authors.affiliation: Department of Hematology, CHU Poitiers, Poitiers xavier.leleu@chu-poitiers.fr.
authors.forename: X
authors.initials: X
authors.lastname: Leleu
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage; D000069286:Bortezomib / Q000008:administration & dosage / Q000009:adverse effects; D003907:Dexamethasone / Q000008:administration & dosage / Q000009:adverse effects; D018572:Disease-Free Survival; D019008:Drug Resistance, Neoplasm / Q000187:drug effects*; D064420:Drug-Related Side Effects and Adverse Reactions / Q000473:pathology; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy* / Q000473:pathology; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000473:pathology; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*
chemical_list: D013792:Thalidomide; D000069286:Bortezomib; D003907:Dexamethasone; C467566:pomalidomide
keywords: continuous therapy; long-term treatment; multiple myeloma; pomalidomide