Bone marrow transplantation is expected to cure blood diseases
Bone marrow transplantation is expected to cure blood diseases
March 20, 2019 Source: Chinese Journal of Science
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];In a recent study, US scientists found that patients receiving bone marrow transplantation from donor cells received double-dose whole-body radiation, which increased the success rate of only “semi-matched†transplants from 50% to nearly 100%. The study was published online March 13 in The Lancet - Hematology. This finding has led to a higher chance of cure for patients with severe and fatal hereditary blood diseases such as sickle cell anemia and beta thalassemia.
“We have succeeded in increasing the cure rate for sickle cell disease and thalassemia to 90%, a result that is very exciting,†said Robert Brodsky, professor of medicine and oncology at Johns Hopkins University School of Medicine and director of hematology. "Bone marrow transplantation technology is not only for patients with fully matched donors. It works equally well for 'semi-matched' patients."
In the late 1980s, Francisco J, associate professor of oncology at the Kimmel Comprehensive Cancer Center in Sydney, Australia. Bolanos-Meade and Brodsky found that bone marrow transplantation has the potential to cure sickle cell disease, and there has been almost no effective treatment for this disease. Patients usually do not live 40 years old. However, few patients have previously used a bone marrow transplantation method that requires a protein that is distributed on the cell surface by bone marrow donors and receptors, that is, human leukocyte antigens are perfectly matched. Brodsky said that in the absence of a perfect match, the receptor would treat the donor cell as a foreign cell and attack it.
Finding a perfectly matched bone marrow donor for this type of patient is difficult because less than 15% of the siblings have a complete match and no genetic defects in the same sickle cell disease, and can find a bloodless relationship that matches exactly. The body of the patient is less than 25%. In 2012, Johns Hopkins University researchers developed a treatment plan that allowed patients to receive bone marrow transplants from only half-matched relatives. Brodsky said this progress has significantly expanded the range of potential donors. In addition, under this transplant protocol, it takes only half of the normal time to generate fresh healthy blood.
To improve the chances of success in these semi-matched transplants, Bolanos-Meade and colleagues tested a new bone marrow transplant program for patients with severe sickle cell disease and beta thalassemia. These two blood diseases, commonly known as hemoglobinopathy, are caused by the same beta globin gene defect. The study enrolled 17 patients, including 12 patients with sickle cell disease and 5 patients with beta thalassemia, with an average age of 16 years. Each patient has a relative who can be a donor of a semi-matched bone marrow transplant.
As in the previous protocol, in order to suppress the immune response of the receptor to the donor bone marrow, all patients received chemotherapy and systemic radiation before transplantation. The new study did not use 200cGy of low-dose radiation as in the previous protocol, but instead used 400cGy of radiation, which is still a relatively small dose of radiation and is well tolerated. After the transplant, all patients took a dose of cyclophosphamide. This drug plays an important role in preventing a potentially fatal disease called graft-versus-host disease, especially in semi-matched transplants.
On the next 30, 60, 180, and 360 days, and each subsequent year, the researchers examined the chimera in the patient's blood, the amount of DNA from the donor, which was a successful transplant. signal. They found that all patients except the one with sickle cell disease showed successful transplantation. Although graft-versus-host disease occurred in five patients, their condition was alleviated.
As of the study release, only 3 patients still need to take immunosuppressive drugs. Among all patients who showed successful transplantation, some cases were greatly relieved, some cases disappeared, and patients with sickle cell disease were no longer suffering from pain. Similarly, patients with beta thalassemia are no longer dependent on blood transfusions.
"These latest findings add a lot of strong evidence to prove the safety and efficacy of a semi-matched bone marrow transplant," said Richard Jones, director of the bone marrow transplant program at the Kimmel Cancer Center. "We have developed more than 1000 semi-matched bone marrow transplants, and Our clinical studies have been successful, and their safety and toxicity are comparable to well-matched bone marrow transplants, so a semi-matched bone marrow transplant should be treated as a treatment for more patients." Jones said for sickle cell disease In the case of thalassemia patients, the use of gene therapy means high-dose (myeloablative) treatment, which is often unbearable, and the transplantation method involves only mild, low-dose treatments with a higher safety factor . (Zhao Xixi)
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