Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Secondary Pulmonary Alveolar Proteinosis with Trisomy-8 Myelodysplastic Syndrome

Abstract 4705

[Background] Pulmonary alveolar proteinosis (PAP) is a rare disorder characterized by abnormal accumulation of alveolar surfactant protein within alveoli. Acquired PAP has been sub-classified into autoimmune and secondary PAP according to the presence of serum anti-granulocyte macrophage colony-stimulating factor (GM-CSF) autoantibody. Hematological diseases including myelodysplastic syndrome (MDS) are the most frequent causes for secondary PAP with unclear pathogenesis independent of anti-GM-CSF antibody.[Objective and method] To assess the clinical effect of HSCT for PAP, we retrospectively analyzed 4 patients with MDS who received allogeneic transplantation at Toranomon Hospital. [Case report] Case 1 is a 35-year-old male with pancytopenia. He was diagnosed with MDS-RA with trisomy 8 abnormality in January 2008. In January 2009, he had productive cough and chest X-ray and CT revealed opaque consolidation in the bilateral lower lung fields. The diagnosis of PAP was made by transbronchial lung biopsy findings. In April 2010, he underwent unrelated bone marrow transplantation (BMT). But idiopathic pneumonia syndrome as a transplant-related complication developed and died on day 55. Case 2 was a 42-year-old female who had a history of aplastic anemia with normal karyotype from March 2007. In March 2009, she had cough and abnormal chest X-ray and CT findings. The diagnosis of PAP was made by bronchoalveolar lavage (BAL) findings. At this time, the diagnosis of MDS-RAEB with trisomy 8 was made. In September 2009, she underwent unrelated cord blood transplantation. But she died by sepsis and pneumonia of Stenotrophomonas maltophilia on day 12. Case 3 was a 58-year-old female with stomatitis who was diagnosed with Behcet's disease in 2001. In May 2001, she developed fever and productive cough. She was diagnosed with PAP by abnormal chest X-ray and BAL findings. In July 2009, she developed pancytopenia, and the diagnosis of MDS-RAEB with trisomy 8 was made. In March 2010, she underwent unrelated BMT. After transplantation, PAP was gradually improved. Case 4 was a 47-year-old male with dyspnea who was diagnosed with PAP by CT and BAL findings. At the same time, he was diagnosed with MDS-RCMD with trisomy 8. In June 2011, he underwent peripheral blood stem cell transplantation from a HLA-identical brother. His transplant clinical course was uneventful and PAP was completely improved by day 42 in CT findings.[Discussion &Conclusion] Case 1 and 2 died of pulmonary complication developed after HSCT, one is pneumonia and another was idiopathic pneumonia syndrome. In case 3 and 4, both transplant clinical course was relatively uneventful and PAP disappeared with the improvement of MDS after HSCT. It is suggested that HSCT might be the effective treatment of secondary PAP with hematological disease, but secondary PAP itself may be the risk of pulmonary complication after HSCT. As we reported the possible association of trisomy 8 MDS with PAP development in 3 cases¹, all 4 MDS cases presented here revealed trisomy 8 abnormality of bone marrow cells.