title: An innovative approach to bone marrow collection and transplantation in a patient with beta-thalassemia major: marrow collection using a perfusion method followed by intra-bone marrow injection of collected bone marrow cells.
abstract: Using small animals (mice and rats) and monkeys, we have found that the combination of bone marrow collection using the perfusion method (PM) and intra-bone marrow-bone marrow transplantation (IBM-BMT) of the collected cells is safe and effective in treating various intractable diseases. Based on these findings, we attempted to apply this method to humans. We report here the first case of a patient (6 years old) with beta-thalassemia major who underwent allogeneic BMT using this new PM + IBM-BMT method. The white blood cell counts of the patient gradually increased to more than 1500/microL by day 47 and continued to increase, reaching the highest level (8600/microL) on day +55. Fluorescence in situ hybridization data on day +33 showed that 98% of the peripheral blood cells were from the donor. Notably, there were no symptoms of graft-versus-host disease (GvHD). However, on day +56, the patient regrettably died of asphyxia resulting from sticky sputum. There was no evidence of infection (in the lung or liver) or GvHD (in the skin) by necropsy. We hope that this case report will help make our new strategies more readily available for the treatment of patients with various intractable diseases.
authors.affiliation: Department of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China.
authors.forename: Chunfu
authors.initials: C
authors.lastname: Li
authors.forename: Yuelin
authors.initials: Y
authors.lastname: He
authors.forename: Xiaoqin
authors.initials: X
authors.lastname: Feng
authors.forename: Muneo
authors.initials: M
authors.lastname: Inaba
authors.forename: Yasushi
authors.initials: Y
authors.lastname: Adachi
authors.forename: Keizo
authors.initials: K
authors.lastname: Takada
authors.forename: Yuming
authors.initials: Y
authors.lastname: Zhang
authors.forename: Yoshihisa
authors.initials: Y
authors.lastname: Yamamoto
authors.forename: Xuedong
authors.initials: X
authors.lastname: Wu
authors.forename: Yunze
authors.initials: Y
authors.lastname: Cui
authors.forename: Masayoshi
authors.initials: M
authors.lastname: Iwasaki
authors.forename: Hiroko
authors.initials: H
authors.lastname: Hisha
authors.forename: Naoki
authors.initials: N
authors.lastname: Hosaka
authors.forename: Mitsuru
authors.initials: M
authors.lastname: Taira
authors.forename: Keizo
authors.initials: K
authors.lastname: Minamino
authors.forename: Yasuhiro
authors.initials: Y
authors.lastname: Suzuki
authors.forename: Keiji
authors.initials: K
authors.lastname: Nakano
authors.forename: Junichi
authors.initials: J
authors.lastname: Fukui
authors.forename: Yusuke
authors.initials: Y
authors.lastname: Ueda
authors.forename: Yasushi
authors.initials: Y
authors.lastname: Koike
authors.forename: Masanobu
authors.initials: M
authors.lastname: Tsuda
authors.forename: Susumu
authors.initials: S
authors.lastname: Ikehara
mesh_terms: D001237:Asphyxia; D001854:Bone Marrow Cells / Q000166:cytology; D016026:Bone Marrow Transplantation / Q000379:methods*; D001842:Bone and Bones; D002648:Child; D017809:Fatal Outcome; D005260:Female; D006801:Humans; D007267:Injections; D007958:Leukocyte Count; D010477:Perfusion / Q000379:methods*; D013183:Sputum; D017086:beta-Thalassemia / Q000628:therapy*