Press release

Tuesday August 27, 2020

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NIH study finds lung function improved or remained the same in adult transplant recipients.

So-called "low-intensity" blood stem cell transplants do not appear to damage the lungs. They may even improve lung function in some patients with sickle cell disease, according to a study of adult transplant patients at the National Institutes of Health.

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Deterioration of lung function and damage to lung tissue is a leading complication and cause of death in people with sickle cell anemia, a blood disorder that can be debilitating. The new study was published in today's issue of the Annals of the American Thoracic Society This study aims to determine whether less intense types of lung transplants, more commonly tolerated by adults, cause additional damage to the lungs.

Dr. Parker Ruhl is a research associate and pulmonologist at the National Institutes of Health. He led the study. The finding should provide reassurance to patients with sickle cell disease who are considering stem cell transplant surgery.

Until recently, the only treatment was bone marrow or blood stem cell transplant. Sickle cell diseaseThe high-dose chemotherapy needed to prepare for the transplant carries health risks. The process also requires that the donor be a genetic match, usually a sibling who does not have sickle cell disease. The procedure involves donating blood stem cells from a healthy donor to produce normal red blood cells to replace the sickle cells. These sickle cells can block blood flow throughout the body. This causes a variety of health problems, including episodes of acute pain, infections, strokes, and acute chest syndrome where the lungs are starved of oxygen.

At least a third of all sickle cell stem cell transplants are done at low intensity, researchers say. Although they may be slightly less effective than conventional transplants in children, adult patients who have suffered more organ damage in the past tend to benefit. They also face a reduced risk of complications. Graft versus host diseaseThis study looked at whether these transplants could offer additional benefits to adults who already have vulnerable lungs.

She and Ruhl's team studied 97 patients with sickle cell disease who underwent low-intensity, nonmyeloablative blood stem cell transfusions between 2004 and 2019 at NIH Clinical Center Bethesda. Participants were followed for up to three years.

The researchers performed several lung tests. One was the forced expiratory volume in one second (FEV1) test, a measure of the air exhaled in the first second of forced expiration. One was a pulmonary diffusion testDLCO, diffusing capacity for carbon monoxide in the lungs (DLCO), measures the amount of oxygen that passes from the lungs into the blood during exhalation. The researchers also performed a 6-minute walk test to measure how far the patient could walk and their oxygen level during that time.

The patients' overall lung function was stable after three years. FEV1 values remained constant after transplantation compared with those before transplantation. This indicates that lung function did not deteriorate over time. Both DLCO and six-minute walk distance improved significantly after transplantation.

Ruhl said that to put the results of the current study in context, larger studies, with longer follow-up periods, and transplant data collected at other clinical centers (including patients who underwent standard organ transplantation) are needed. She and her team are continuing to follow the NIH patient and will report longer-term results at five and 10 years.

The U.S. Food and Drug Administration will approve the product in December 2023. Two genetic treatments Using a patient's own stem cells in the treatment of sickle cell disease. The researchers hope to use the same techniques in their study to assess lung function when developing new genetic treatments.

The National Heart, Lung, and Blood Institute of the NIH (grant 1U01HL156620-01) and the Divisions of Intramural Research of the National Institute of Allergy and Infectious Diseases of the NIH (projects HL006211-8 and HL006007-6 and project HL006211-18) funded this study in part.

The National Heart, Lung, and Blood Institute: NHLBI, a global leader, conducts and supports research on heart, blood, lung and sleep diseases that improves public health care and saves lives. Visit the website for more information. www.nhlbi.nih.gov.

The National Institutes of Health: The NIH is the medical research agency of the United States Department of Health and Human Services. It includes 27 institutes and centers. The NIH, the nation's medical research agency, is the primary federal agency that conducts and supports basic, translational, and clinical medical research. It also studies the causes, treatment, and cures of common and rare diseases. Visit the NIH for more information about its programs and services. www.nih.gov.

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