title: Mannose-binding lectin (MBL) therapy in an MBL-deficient patient with severe cystic fibrosis lung disease.
abstract: Deficiency of mannose-binding lectin has been shown to be a risk factor for cystic fibrosis (CF) patients. We, therefore, decided to treat a patient with CF, mannose-binding lectin deficiency, severe bronchopulmonary Pseudomonas aeruginosa infection, and rapid deterioration of lung function with purified mannose-binding lectin in an attempt to ameliorate the course of the lung disease. The mannose-binding lectin used originated from pooled human donor plasma and was given as an intravenous infusion twice a week for a period of 3 months. The patients's clinical condition was stabilized during the treatment period, but was not improved. No adverse events were observed. However, the lung function assessed as percent forced expiratory volume in 1 sec (FEV1%) and percent forced vital capacirt (FVC%) correlated significantly with the mannose-binding serum lectin levels (rho=+0.68, P=0.008, and rho=+0.73, P=0.004). Additionally, an inverse correlation with the acute phase-reactant C-reactive protein and the proinflammatory cytokine IL-6 was observed (rho=-0.49, P=0.007 and rho=-0.41, P=0.04, respectively). The results emphasize the importance of mannose-binding lectin as a secondary disease modifier in CF. Moreover, purified mannose-binding lectin can safely be administered to chronically ill patients, and may be a potential treatment in CF and other diseases in which mannose-binding lectin deficiency plays a pathophysiological role.
authors.affiliation: Tissue Typing Laboratory, Department of Clinical Immunology, National University Hospital (Rigshospitalet), Copenhagen, Denmark.
authors.forename: Peter
authors.initials: P
authors.lastname: Garred
authors.forename: Tacjana
authors.initials: T
authors.lastname: Pressler
authors.forename: Susanne
authors.initials: S
authors.lastname: Lanng
authors.forename: Hans O
authors.initials: HO
authors.lastname: Madsen
authors.forename: Claus
authors.initials: C
authors.lastname: Moser
authors.forename: Inga
authors.initials: I
authors.lastname: Laursen
authors.forename: Flemming
authors.initials: F
authors.lastname: Balstrup
authors.forename: Claus
authors.initials: C
authors.lastname: Koch
authors.forename: Christian
authors.initials: C
authors.lastname: Koch
mesh_terms: D000328:Adult; D000483:Alleles; D002097:C-Reactive Protein / Q000378:metabolism; D002352:Carrier Proteins / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*; D037222:Collectins; D003550:Cystic Fibrosis / Q000150:complications / Q000188:drug therapy* / Q000235:genetics; D018450:Disease Progression; D004359:Drug Therapy, Combination; D017809:Fatal Outcome; D005260:Female; D005541:Forced Expiratory Volume; D006801:Humans; D015850:Interleukin-6 / Q000378:metabolism; D009154:Mutation; D011552:Pseudomonas Infections / Q000150:complications; D013147:Spirometry; D014409:Tumor Necrosis Factor-alpha / Q000378:metabolism; D014797:Vital Capacity
chemical_list: D002352:Carrier Proteins; D037222:Collectins; D015850:Interleukin-6; D014409:Tumor Necrosis Factor-alpha; D002097:C-Reactive Protein