Premium
Successful bone marrow transplantation with reduced intensity conditioning in a patient with delayed‐onset adenosine deaminase deficiency
Author(s) -
Kanegane Hirokazu,
Taneichi Hiromichi,
Nomura Keiko,
Wada Taizo,
Yachie Akihiro,
Imai Kohsuke,
Ariga Tadashi,
Santisteban Ines,
Hershfield Michael S.,
Miyawaki Toshio
Publication year - 2013
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/j.1399-3046.2012.01762.x
Subject(s) - medicine , adenosine deaminase deficiency , fludarabine , neutropenia , adenosine deaminase , busulfan , pediatrics , transplantation , hematopoietic stem cell transplantation , adenosine , chemotherapy , cyclophosphamide
Kanegane H, Taneichi H, Nomura K, Wada T, Yachie A, Imai K, Ariga T, Santisteban I, Hershfield MS, Miyawaki T. Successful bone marrow transplantation with reduced intensity conditioning in a patient with delayed‐onset adenosine deaminase deficiency. Abstract: In this case report, we describe successful BMT with RIC in a patient with delayed‐onset ADA deficiency. A three‐yr‐old Japanese boy was diagnosed with delayed‐onset ADA deficiency because of recurrent bronchitis, bronchiectasia, and lymphopenia. In addition, autoimmune thyroiditis and neutropenia were present. At four yr of age, he underwent BMT with a RIC regimen, including busulfan and fludarabine, from an HLA‐identical healthy sister. Engraftment after BMT was uneventful without GVHD. Decreased ADA levels in blood immediately increased following BMT, and the patient was disease‐free 13 months after BMT. These results suggest that BMT with RIC may sufficiently restore immune regulation in delayed‐onset ADA deficiency. A longer follow‐up period is needed to confirm these observations.