Severe chronic primary neutropenia in adults: report on a series of 108 patients.
Severe chronic primary neutropenia (CPN) is a rare entity, and long-term outcome and risk factors for infections in severe CPN adults have not been described to date. We report the characteristics and outcomes of 108 severe adult CPN patients enrolled in a multi-institutional observational study. Severe CPN adults were mostly female (78%), and median age at diagnosis was 28.3 years. Diagnosis was fortuitous in 62% of cases. The median absolute neutrophil count (ANC) at diagnosis was 0.4 × 10(9)/L, and median ANC without granulocyte colony-stimulating factor (G-CSF) during follow-up was 0.5 × 10(9)/L. Twenty-three of 66 (34.8%) evaluable patients had neutrophil autoantibodies, and 6 of 47 (12.8%) a T-cell clone. The presence of neutrophil autoantibodies or T-cell clone was not associated with any specific clinical or biological characteristics. No death or hematologic malignancies occurred, and 44 severe bacterial infections were reported in 27 patients with a median follow-up of 8.3 years. Fifty patients received G-CSF either sporadically (n = 24) or continuously (n = 26) and responded (96%). Nineteen patients received immunosuppressive therapies: overall response (OR) was 41%, and median duration of response was 3 months. At diagnosis, the only predictive factor for the occurrence of severe bacterial infections was an ANC count below 0.2 × 10(9)/L (OR, 0.76). Severe CPN in adults is characterized by a female predominance and a benign outcome with a low rate of severe bacterial infections and no secondary malignancies. G-CSF is efficient and well tolerated but is not required in a majority of patients.
Service d'Hématologie-Greffe, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Paris, France;
Flore
F
Sicre de Fontbrune
Département d'Hématologie Clinique, Hôpital Universitaire de Rennes, Rennes, France, and INSERM U1414-CIC, Université Rennes I;
Aline
A
Moignet
Service d'Hématologie Pédiatrique, Hôpital Trousseau, Assistance Publique Hôpitaux de Paris, Paris, France;
Blandine
B
Beaupain
Service d'Hématologie Adulte, Hôpital Necker-Enfants Malades, Assistance Publique Hôpitaux de Paris, Paris, France;
Felipe
F
Suarez
Service d'Immunologie Clinique, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Paris, France;
Lionel
L
Galicier
Service d'Hématologie-Greffe, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Paris, France;
Gérard
G
Socié
Service d'Hématologie Adulte, Hôpital Necker-Enfants Malades, Assistance Publique Hôpitaux de Paris, Paris, France;
Bruno
B
Varet
Service d'Hématologie, Hôpital Saint Antoine, Assistance Publique Hôpitaux de Paris, Paris, France;
Paul
P
Coppo
Service de Médecine Interne and.
Marc
M
Michel
Service d'Hématologie, Hôpital Henri Mondor, Assistance Publique Hôpitaux de Paris, Paris, France;
Cécile
C
Pautas
Service d'Immunologie Clinique, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Paris, France;
Eric
E
Oksenhendler
Service d'Hématologie Adulte, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Paris, France;
Etienne
E
Lengline
Service d'Hématologie, Centre Hospitalier Régional Universitaire de Lille, Lille, France;
Louis
L
Terriou
Service d'Hématologie, Hôpital Hôtel-Dieu, Centre Hospitalier Régional Universitaire de Nantes, Nantes, France;
Philippe
P
Moreau
Service d'Hématologie Clinique, Centre Hospitalier Universitaire de Caen, Caen, France;
Sylvain
S
Chantepie
Service d'Hématologie, Hôpital Saint Antoine, Assistance Publique Hôpitaux de Paris, Paris, France;
Nicole
N
Casadevall
Service de Médecine Interne, Hôpital Kremlin-Bicêtre, Assistance Publique Hôpitaux de Paris, Kremlin-Bicêtre, France;
Jean Marie
JM
Michot
Service des Maladies du Sang, Centre Hospitalier Universitaire Angers, Angers, France;
Martine
M
Gardembas
Service d'Hématologie, Hôpital Lyon-Sud, Hospices Civiles de Lyon, Lyon, France;
Mauricette
M
Michallet
Laboratoire d'Immunologie Leuco-plaquettaire et d'Histocompatibilité, Hôpital Henri Mondor, Etablissement Français du Sang d'Ile de France, Créteil, France;
Laure
L
Croisille
Laboratoire d'Immunologie, Institut de Biologie, Centre Hospitalier Universitaire de Nantes, Nantes, France; and.
Marie
M
Audrain
Département de Génétique, Hôpital de la Pitié-Salpêtrière, Université Pierre et Marie Curie, Assistance Publique Hôpitaux de Paris, Paris, France.
Christine
C
Bellanné-Chantelot
Service d'Hématologie Pédiatrique, Hôpital Trousseau, Assistance Publique Hôpitaux de Paris, Paris, France;
Jean
J
Donadieu
Département d'Hématologie Clinique, Hôpital Universitaire de Rennes, Rennes, France, and INSERM U1414-CIC, Université Rennes I;
Thierry
T
Lamy
D000328:Adult; D001323:Autoantibodies / Q000097:blood; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D009503:Neutropenia / Q000097:blood* / Q000188:drug therapy / Q000473:pathology*
D001323:Autoantibodies; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor