anemia, CDA），是一種罕見的血液疾病，其中身體不能產生足夠的紅細胞，引起器官的器官損傷和早期死亡，患者只能不斷的到醫院進行輸血才能存活。而最近伊利諾伊大學醫院與健康科學（University of Illinois Hospital & Health Sciences System）的醫師使用了幹細胞移植，並避免使用大劑量化學療法和放射線，成為首例成年後先天性造血性貧血患者的治療成功。
The transplant technique is unique, because
it allows a donor's cells to gradually take over a patient's bone marrow
without using toxic agents to eliminate a patient's cells prior to the
Dr. Damiano Rondelli, the Michael Reese
Professor of Hematology at the University of Illinois at Chicago, says the
protocol can be used even in patients with a long history of disease and some
organ damage because of the minimal use of chemotherapy.
"For many adult patients with a blood
disorder, treatment options have been limited because they are often not sick
enough to qualify for a risky procedure, or they are too sick to tolerate the
toxic drugs used alongside a standard transplant," said Rondelli, who is
also division chief of hematology and oncology and director of the stem cell transplant
program at UI Health.
"This procedure gives some adults the
option of a stem cell transplant which was not previously available."
For more than 30 years, Northbrook,
Illinois, resident David Levy's only course of treatment for CDA was regular
blood transfusions to ensure his organs and tissues received enough oxygen.
Levy was 24 when the pain became so severe he had to withdraw from graduate
"I spent the following years doing
nothing -- no work, no school, no social contact -- because all I could focus
on was managing my pain and getting my health back on track," Levy said.
By age 32, Levy required transfusions every
two to three weeks; had lost his spleen; had an enlarged liver; and was
suffering severely from fatigue, heart palpitations and iron poisoning, a side
effect of regular blood transfusions.
"It was bad," Levy said. "I
had been through enough pain. I was angry and depressed, and I wanted a cure.
That's why I started emailing Dr. Rondelli."
Rondelli says that because of Levy's range
of illnesses and inability to tolerate chemotherapy and radiation, several
institutions had denied him the possibility of a stem cell transplant. UI
Health's advances in curing sickle cell patients opened up a new possibility.
Rondelli performed Levy's transplant in 2014.
"The transplant was hard, and I had
some complications, but I am back to normal now," said Levy, now 35.
"I still have some pain and some lingering issues from the years my
condition was not properly managed, but I can be independent now. That is the most
important thing to me."
Levy is finishing his doctorate in
psychology and running group therapy sessions at a behavioral health hospital.
Rondelli says the potential of this
approach to stem cell transplantation is very promising.
"The use of this transplant protocol
may represent a safe therapeutic strategy to treat adult patients with many
types of congenital anemias -- perhaps the only possible cure," Rondelli
This case report is published in a letter
to the editor in the journal Bone Marrow Transplantation.
of Illinois at Chicago. " First patient
cured of rare blood disorder. " ScienceDaily, 20 March, 2017.
參考文獻：A Oh, P R
Patel et al., Non-myeloablative
allogeneic stem cell transplant with post-transplant cyclophosphamide cures the
first adult patient with congenital dyserythropoietic anemia. Bone Marrow Transplantation, 2017. DOI: