title: Ofatumumab in refractory chronic lymphocytic leukemia: experience through the French early access program.
abstract: BACKGROUND
  The Autorisation Temporaire d'Utilisation (ATU) is an early access program available in France for drugs aimed at treating severe diseases not yet covered by a marketing authorization, for patients without any other therapeutic option and who cannot be included in a clinical trial.
  
  
  METHODS
  This report presents the use of single-agent ofatumumab in 30 patients with advanced chronic lymphocytic leukemia (CLL) in the French ATU program.
  
  
  RESULTS
  These very-high-risk patients had received multiple previous treatments (median = 6), and most had disease that was fludarabine-refractory or alemtuzumab-refractory (or both) or was unsuitable for alemtuzumab treatment. In the intent-to-treat analysis, the overall response rate was 47% (4 of 30, complete response; 10 of 30, partial response). Of 13 patients with 17p deletion, 6 displayed response to ofatumumab, including 2 complete responses. Treatment was well tolerated, with 17 grade 3 or 4 adverse events; 4 cases of grade 3 or 4 infusion reactions were reported, with favorable immediate outcome. Among nonhematologic complications, infections were the most frequent.
  
  
  CONCLUSIONS
  The results confirm the efficacy and acceptable tolerability profile of ofatumumab as a single agent in severely ill patients with CLL. Attention should be paid to possible early infusion reactions to ofatumumab, as well as to the risk of infection.
authors.affiliation: Unité Fonctionnelle Hémopathies Lymphoïdes, CHU Henri Mondor, Créteil, France. Electronic address: jehan.dupuis@hmn.aphp.fr.
authors.forename: J
authors.initials: J
authors.lastname: Dupuis
authors.affiliation: HDJ Hématologie, Hôpital Saint Louis, Paris, France.
authors.forename: P
authors.initials: P
authors.lastname: Brice
authors.affiliation: Service d'Hématologie, CHU d'Angers, Angers, France.
authors.forename: S
authors.initials: S
authors.lastname: François
authors.affiliation: Service d'Hématologie, CHU Purpan, Toulouse, France.
authors.forename: L
authors.initials: L
authors.lastname: Ysebaert
authors.affiliation: Service d'Hématologie Clinique, CHU Pontchaillou, Rennes, France.
authors.forename: S
authors.initials: S
authors.lastname: de Guibert
authors.affiliation: Unité de Recherche Clinique, Groupe Hospitalier Paris Seine Saint Denis, Bobigny, France.
authors.forename: V
authors.initials: V
authors.lastname: Levy
authors.affiliation: Département d'Hématologie, Centre Henri Becquerel, Rouen, France.
authors.forename: S
authors.initials: S
authors.lastname: Leprêtre
authors.affiliation: Département d'Hématologie Clinique, Hôpital Pitié-Salpétrière, Paris, France.
authors.forename: S
authors.initials: S
authors.lastname: Choquet
authors.affiliation: Service d'hématologie clinique et thérapie cellulaire, Hôpital Haut Lévèque, CHU de Bordeaux, Pessac, France.
authors.forename: M S
authors.initials: MS
authors.lastname: Dilhuydy
authors.affiliation: Service d'Oncologie et d'Hématologie, Hôpitaux Universitaires de Strasbourg, Strasbourg, France.
authors.forename: L
authors.initials: L
authors.lastname: Fornecker
authors.affiliation: Département d'Hématologie Clinique, Hôpital Pitié-Salpétrière, Paris, France.
authors.forename: V
authors.initials: V
authors.lastname: Morel
authors.affiliation: Service d'Hématologie Clinique, CHRU de Brest, Brest, France.
authors.forename: A
authors.initials: A
authors.lastname: Tempescul
mesh_terms: D000368:Aged; D000911:Antibodies, Monoclonal / Q000008:administration & dosage* / Q000009:adverse effects; D061067:Antibodies, Monoclonal, Humanized; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects; D019008:Drug Resistance, Neoplasm; D005260:Female; D005602:France; D006801:Humans; D015451:Leukemia, Lymphocytic, Chronic, B-Cell / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000970:Antineoplastic Agents; C527517:ofatumumab
keywords: CD20; CLL; Ofatumumab; Relapsed/refractory; Temporary use authorization