Hematopoietic stem cell transplantation for pulmonary alveolar proteinosis associated with primary immunodeficiency disease
Pulmonary alveolar proteinosis (PAP) is a rare disorder that is characterized by the excessive accumulation of surfactant-like materials in the alveoli, leading to hypoxemic respiratory failure. We describe two Japanese infants with PAP associated with hypogammaglobulinemia and monocytopenia. These patients may have underlying primary immunodeficiency (PID) and were successfully treated with allogeneic hematopoietic stem cell transplantation (HSCT). This report indicates that allogeneic HSCT may provide a curative treatment for PAP associated with PID.
KeywordsPulmonary alveolar proteinosis Hematopoietic stem cell transplantation Primary immunodeficiency Granulocyte–macrophage colony stimulating factor Bronchoalveolar lavage
The authors would like to thank Dr. Hisaya Hasegawa in Tokyo Women’s Medical University Medical Center East and Dr. Kazutoshi Cho in Hokkaido University for performing BAL, and Dr. Koh Nakata in Niigata University for measuring anti-GM-CSF antibody.
Compliance with ethical standards
Funding was supported by Ministry of Education, Culture, Sports, Science, and Technology of Japan, and Ministry of Health, Labour, and Welfare of Japan.
Conflict of interest
The authors declare that there is no conflict of interest.
- 24.Nishinakamura R, Wiler R, Dirksen U, Morikawa Y, Arai K, Miyajima A, et al. The pulmonary alveolar proteinosis in granulocyte macrophage colony-stimulating factor/interleukins 3/5 beta c receptor-deficient mice is reversed by bone marrow transplantation. J Exp Med. 1996;183:2657–62.CrossRefPubMedGoogle Scholar