Half of patients in a trial have safely stopped immunosuppressant medication following a modified blood stem-cell transplant for severe sickle cell disease, according to a study in the
The transplant done in the study reversed sickle cell disease in nearly all the patients. Despite having both donor stem-cells and their own cells in their blood, the patients stopped the immunosuppressant medication without experiencing rejection or graft-versus-host disease, in which donor cells attack the recipient. Both are common, serious side effects of transplants.
Image of blood from a sickle cell patient Blood drawn from a sickle cell patient. Source: NIH Molecular and Clinical Hematology Branch "Typically, stem-cell recipients must take immunosuppressants all their lives," said
In sickle cell disease (SCD) sickle-shaped cells block blood flow. It can cause severe pain, organ damage and stroke. The only cure is a blood stem-cell, or bone marrow, transplant. The partial transplant performed in the study is much less toxic than the standard "full" transplant, which uses high doses of chemotherapy to kill all of the patient's marrow before replacing it with donor marrow. Several patients in the study had less than half of their marrow replaced.
Immunosuppressant medication reduces immune system strength and can cause serious side effects such as infection and joint swelling. In this study, 15 of 30 adults stopped taking the medication under careful supervision one year after transplant and still had not experienced rejection or graft-versus-host disease at a median follow up of 3.4 years.
"Side effects caused by immunosuppressants can endanger patients already weakened by years of organ damage from sickle cell disease," said
Image of blood from a sickle cell patient after treatment Blood drawn from a sickle cell patient after stem-cell transplant. Source: NIH Molecular and Clinical Hematology Branch "One of the most debilitating effects of sickle cell disease is the often relentless pain," added Dr. Hsieh. "Following the transplant, we saw a significant decrease in hospitalizations and narcotics to control that pain."
The partial transplant used donor stem-cells from healthy siblings. It effectively reversed SCD in 26 of 30 patients and allowed them to achieve stable mixed donor chimerism, a condition in which a person has two genetically distinct cell types in the blood. The study includes patients from an NIH study reported in 2009, in which partial stem-cell transplants reversed SCD in 9 of 10 people.
"The devastating complications associated with sickle cell disease can deeply affect quality of life, ability to work and long-term well-being," said NIDDK Director
People with sickle cell disease interested in joining NIH blood stem-cell transplant studies may call 1-800-411-1222 or visit http://www.clinicaltrials.gov for more information.
More information on sickle cell disease is available at: http://www.nhlbi.nih.gov/health/dci/Diseases/Sca/SCA_WhatIs.html and http://diabetes.niddk.nih.gov/dm/pubs/hemovari-A1C.
The NIDDK, part of the NIH, conducts and supports basic and clinical research and research training on some of the most common, severe and disabling conditions affecting Americans. The institute's research interests include: diabetes and other endocrine and metabolic diseases; digestive diseases, nutrition, and obesity; and kidney, urologic and hematologic diseases. For more information, visit http://www.niddk.nih.gov.
Part of the NIH, the National Heart, Lung, and
The NIH Clinical Center (CC) provided clinical laboratory and transfusion medicine support and care for stem-cell donors and recipients. The CC is the clinical research hospital for the NIH. Through clinical research, physician-investigators translate laboratory discoveries into better treatments, therapies and interventions to improve the nation's health. For more information, visit http://clinicalcenter.nih.gov.
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