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Chest. 2014 Apr;145(4):729-737. doi: 10.1378/chest.13-0603.

Duration of benefit in patients with autoimmune pulmonary alveolar proteinosis after inhaled granulocyte-macrophage colony-stimulating factor therapy.

Author information

1
Niigata University Medical and Dental Hospital, Niigata.
2
National Hospital Organization (NHO) Kinki-Chuo Chest Medical Center, Osaka.
3
Niigata University Graduate School of Medical and Dental Sciences, Niigata.
4
Department of Respirology, Graduate School of Medicine, Chiba University, Chiba.
5
Division of Respiratory Medicine, National Center for Global Health and Medicine, Tokyo.
6
Department of Respiratory Medicine, Tohoku University Medical School, Sendai.
7
Juzenkai Hospital, Nagasaki; Institute of Tropical Medicine, Nagasaki University, Nagasaki.
8
Kitasato University School of Allied Health Sciences, Kanagawa.
9
NHO Yamaguchi-Ube Medical Center, Ube; Kurashiki Municipal Kojima Hospital, Kurashiki.
10
Niigata University Graduate School of Medical and Dental Sciences, Niigata; Department of Respiratory Medicine, Tokyo Medical University, Tokyo.
11
Department of Respiratory Medicine, Kyorin University School of Medicine, Tokyo.
12
Department of Respiratory Medicine, Nippon Medical University School of Medicine, Tokyo.
13
Institute of Tropical Medicine, Nagasaki University, Nagasaki.
14
First Department of Medicine, Hokkaido University School of Medicine, Sapporo.
15
Department of Respiratory Medicine, Tokyo Medical University Hachioji Medical Center, Tokyo.
16
Division of Respiratory Medicine and Allergology, Department of Medicine, Aichi Medical University School of Medicine, Aichi, Japan.
17
Niigata University Medical and Dental Hospital, Niigata. Electronic address: radical@med.niigata-u.ac.jp.

Abstract

BACKGROUND:

Treatment of autoimmune pulmonary alveolar proteinosis (aPAP) by subcutaneous injection or inhaled therapy of granulocyte-macrophage colony-stimulating factor (GM-CSF) has been demonstrated to be safe and efficacious in several reports. However, some reports of subcutaneous injection described transient benefit in most instances. The durability of response to inhaled GM-CSF therapy is not well characterized.

METHODS:

To elucidate the risk factors for recurrence of aPAP after GM-CSF inhalation, 35 patients were followed up, monitoring for the use of any additional PAP therapies and disease severity score every 6 months. Physiologic, serologic, and radiologic features of the patients were analyzed for the findings of 30-month observation after the end of inhalation therapy.

RESULTS:

During the observation, 23 patients remained free from additional treatments, and twelve patients required additional treatments. There were no significant differences in age, sex, symptoms, oxygenation indexes, or anti-GM-CSF antibody levels at the beginning of treatment between the two groups. Baseline vital capacity (% predicted, %VC) were higher among those who required additional treatment (P<.01). Those patients not requiring additional treatment maintained the improved disease severity score initially achieved. A significant difference in the time to additional treatment between the high %VC group (%VC≥80.5) and the low %VC group was seen by a Kaplan-Meier analysis and a log-rank test (P<.0005).

CONCLUSIONS:

These results demonstrate that inhaled GM-CSF therapy sustained remission of aPAP in more than one-half of cases, and baseline %VC might be a prognostic factor for disease recurrence.

TRIAL REGISTRY:

ISRCTN Register and JMACCT Clinical Trial Registry; No.: ISRCTN18931678 and JMAIIA00013; URL: http://www.isrctn.org and http://www.jmacct.med.or.jp.

PMID:
24158247
DOI:
10.1378/chest.13-0603
[Indexed for MEDLINE]

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