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.
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.
Li
Chunfu
C
Department of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China.
He
Yuelin
Y
Feng
Xiaoqin
X
Inaba
Muneo
M
Adachi
Yasushi
Y
Takada
Keizo
K
Zhang
Yuming
Y
Yamamoto
Yoshihisa
Y
Wu
Xuedong
X
Cui
Yunze
Y
Iwasaki
Masayoshi
M
Hisha
Hiroko
H
Hosaka
Naoki
N
Taira
Mitsuru
M
Minamino
Keizo
K
Suzuki
Yasuhiro
Y
Nakano
Keiji
K
Fukui
Junichi
J
Ueda
Yusuke
Y
Koike
Yasushi
Y
Tsuda
Masanobu
M
Ikehara
Susumu
S
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*