Early and prolonged intravenous immunoglobulin replacement therapy in childhood agammaglobulinemia: a retrospective survey of 31 patients.
OBJECTIVE
To evaluate the outcome of children who received prolonged intravenous immunoglobulin (IVIg) replacement therapy early in life for X-linked agammaglobulinemia (XLA).


METHODS
We performed a retrospective study of the clinical features and outcome of patients with genetic and/or immunologic results consistent with XLA. Patients receiving IVIg replacement therapy within 3 months of the diagnosis and for at least 4 years between 1982 and 1997 were included.


RESULTS
Thirty-one patients began receiving IVIg replacement therapy at a median age of 24 months and were followed up for a median time of 123 months. IVIg was given at doses >0.25 g/kg every 3 weeks, and mean individual residual IgG levels ranged from 500 to 1140 mg/dL (median, 700 mg/dL). During IVIg replacement, the incidence of bacterial infections requiring hospitalization fell from 0.40 to 0.06 per patient per year (P <. 001). However, viral or unidentified infections still developed, including enteroviral meningoencephalitis (n = 3) causing death in one patient, exudative enteropathy (n = 3), and aseptic arthritis (n = 1). At last follow-up, 30 patients were alive at a median age of 144 months (range, 58 to 253 months). Among 23 patients who were evaluated by respiratory function tests and computed tomography, 3 had an obstructive syndrome, 6 had bronchiectasis, and 20 had chronic sinusitis.


CONCLUSIONS
Early IVIg replacement therapy achieving residual IgG levels >500 mg/dL is effective in preventing severe acute bacterial infections and pulmonary insufficiency. More intensive therapy may be required to fully prevent the onset of bronchiectasis, chronic sinusitis, and nonbacterial infections, particularly enteroviral infections, in all cases.
Quartier
P
P
Pediatric Immunology and Hematology Unit and Institut National de la Santé et de la Recherche Médicale U429, Pediatric Pneumology Unit, Hôpital Necker-Enfants Malades, 149, rue de Sévres, Paris, France.
Debré
M
M
De Blic
J
J
de Sauverzac
R
R
Sayegh
N
N
Jabado
N
N
Haddad
E
E
Blanche
S
S
Casanova
J L
JL
Smith
C I
CI
Le Deist
F
F
de Saint Basile
G
G
Fischer
A
A
D000361:Agammaglobulinemia / Q000150:complications / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D005500:Follow-Up Studies; D008040:Genetic Linkage; D006801:Humans; D007070:Immunoglobulin A / Q000097:blood; D007074:Immunoglobulin G / Q000097:blood; D007075:Immunoglobulin M / Q000097:blood; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use*; D016130:Immunophenotyping; D007223:Infant; D007239:Infections / Q000209:etiology; D012189:Retrospective Studies; D014960:X Chromosome
D007070:Immunoglobulin A; D007074:Immunoglobulin G; D007075:Immunoglobulin M; D016756:Immunoglobulins, Intravenous