Literature DB >> 29185156

Hematopoietic stem cell transplantation for pulmonary alveolar proteinosis associated with primary immunodeficiency disease.

Mari Tanaka-Kubota1, Koji Shinozaki2, Satoshi Miyamoto1, Masakatsu Yanagimachi1, Tsubasa Okano1, Noriko Mitsuiki1, Masahiro Ueki3, Masafumi Yamada3, Kohsuke Imai4, Masatoshi Takagi4, Kazunaga Agematsu2,5, Hirokazu Kanegane6, Tomohiro Morio1.   

Abstract

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.

Entities:  

Keywords:  Bronchoalveolar lavage; Granulocyte–macrophage colony stimulating factor; Hematopoietic stem cell transplantation; Primary immunodeficiency; Pulmonary alveolar proteinosis

Mesh：

Substances：

Year:  2017        PMID： 29185156     DOI： 10.1007/s12185-017-2375-1

Source DB:  PubMed          Journal:  Int J Hematol        ISSN： 0925-5710            Impact factor:   2.490


  25 in total

1.  A mutation in the surfactant protein C gene associated with familial interstitial lung disease.

Authors:  L M Nogee; A E Dunbar; S E Wert; F Askin; A Hamvas; J A Whitsett
Journal:  N Engl J Med       Date:  2001-02-22       Impact factor: 91.245

2.  Pulmonary alveolar proteinosis in patients with adenosine deaminase deficiency.

Authors:  Eyal Grunebaum; Ernest Cutz; Chaim M Roifman
Journal:  J Allergy Clin Immunol       Date:  2012-03-10       Impact factor: 10.793

3.  Human pulmonary alveolar proteinosis associated with a defect in GM-CSF/IL-3/IL-5 receptor common beta chain expression.

Authors:  U Dirksen; R Nishinakamura; P Groneck; U Hattenhorst; L Nogee; R Murray; S Burdach
Journal:  J Clin Invest       Date:  1997-11-01       Impact factor: 14.808

4.  BAL findings in a patient with pulmonary alveolar proteinosis successfully treated with GM-CSF.

Authors:  O D Schoch; U Schanz; M Koller; K Nakata; J F Seymour; E W Russi; A Boehler
Journal:  Thorax       Date:  2002-03       Impact factor: 9.139

5.  Surfactant proteins in pulmonary alveolar proteinosis in adults.

Authors:  F Brasch; J Birzele; M Ochs; S H Guttentag; O D Schoch; A Boehler; M F Beers; K M Müller; S Hawgood; G Johnen
Journal:  Eur Respir J       Date:  2004-09       Impact factor: 16.671

6.  GM-CSF autoantibodies and neutrophil dysfunction in pulmonary alveolar proteinosis.

Authors:  Kanji Uchida; David C Beck; Takashi Yamamoto; Pierre-Yves Berclaz; Shuichi Abe; Margaret K Staudt; Brenna C Carey; Marie-Dominique Filippi; Susan E Wert; Lee A Denson; Jonathan T Puchalski; Diane M Hauck; Bruce C Trapnell
Journal:  N Engl J Med       Date:  2007-02-08       Impact factor: 91.245

7.  Clinical significance of anti-GM-CSF antibodies in idiopathic pulmonary alveolar proteinosis.

Authors:  F-C Lin; G-D Chang; M-S Chern; Y-C Chen; S-C Chang
Journal:  Thorax       Date:  2006-03-03       Impact factor: 9.139

8.  The pulmonary alveolar proteinosis in granulocyte macrophage colony-stimulating factor/interleukins 3/5 beta c receptor-deficient mice is reversed by bone marrow transplantation.

Authors:  R Nishinakamura; R Wiler; U Dirksen; Y Morikawa; K Arai; A Miyajima; S Burdach; R Murray
Journal:  J Exp Med       Date:  1996-06-01       Impact factor: 14.307

9.  Characterization of CSF2RA mutation related juvenile pulmonary alveolar proteinosis.

Authors:  Jenna Hildebrandt; Ebru Yalcin; Hans-Georg Bresser; Guzin Cinel; Monika Gappa; Alireza Haghighi; Nural Kiper; Soheila Khalilzadeh; Karl Reiter; John Sayer; Nicolaus Schwerk; Anke Sibbersen; Sabine Van Daele; Georg Nübling; Peter Lohse; Matthias Griese
Journal:  Orphanet J Rare Dis       Date:  2014-11-26       Impact factor: 4.123

10.  Familial pulmonary alveolar proteinosis caused by mutations in CSF2RA.

Authors:  Takuji Suzuki; Takuro Sakagami; Bruce K Rubin; Lawrence M Nogee; Robert E Wood; Sarah L Zimmerman; Teresa Smolarek; Megan K Dishop; Susan E Wert; Jeffrey A Whitsett; Gregory Grabowski; Brenna C Carey; Carrie Stevens; Johannes C M van der Loo; Bruce C Trapnell
Journal:  J Exp Med       Date:  2008-10-27       Impact factor: 14.307
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  4 in total

Review 1.  Pulmonary alveolarproteinosis in children.

Authors:  Andrew Bush; Rishi Pabary
Journal:  Breathe (Sheff)       Date:  2020-06

2.  Hematopoietic Cell Transplantation with Reduced Intensity Conditioning Using Fludarabine/Busulfan or Fludarabine/Melphalan for Primary Immunodeficiency Diseases.

Authors:  Akira Nishimura; Yuki Aoki; Yasuyoshi Ishiwata; Takuya Ichimura; Junichi Ueyama; Yuta Kawahara; Takahiro Tomoda; Maiko Inoue; Kazuaki Matsumoto; Kento Inoue; Haruka Hiroki; Shintaro Ono; Motoi Yamashita; Tsubasa Okano; Mari Tanaka-Kubota; Miho Ashiarai; Satoshi Miyamoto; Reiji Miyawaki; Chika Yamagishi; Mari Tezuka; Teppei Okawa; Akihiro Hoshino; Akifumi Endo; Masato Yasuhara; Takahiro Kamiya; Noriko Mitsuiki; Toshiaki Ono; Takeshi Isoda; Masakatsu Yanagimachi; Daisuke Tomizawa; Masayuki Nagasawa; Shuki Mizutani; Michiko Kajiwara; Masatoshi Takagi; Hirokazu Kanegane; Kohsuke Imai; Tomohiro Morio
Journal:  J Clin Immunol       Date:  2021-02-01       Impact factor: 8.317

3.  Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis.

Authors:  Miriam Hetzel; Elena Lopez-Rodriguez; Adele Mucci; Ariane Hai Ha Nguyen; Takuji Suzuki; Kenjiro Shima; Theresa Buchegger; Sabine Dettmer; Thomas Rodt; Jens P Bankstahl; Punam Malik; Lars Knudsen; Axel Schambach; Gesine Hansen; Bruce C Trapnell; Nico Lachmann; Thomas Moritz
Journal:  Haematologica       Date:  2019-07-09       Impact factor: 9.941

Review 4.  Beyond "Big Eaters": The Versatile Role of Alveolar Macrophages in Health and Disease.

Authors:  Miriam Hetzel; Mania Ackermann; Nico Lachmann
Journal:  Int J Mol Sci       Date:  2021-03-24       Impact factor: 5.923
  4 in total

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