Novel parental-donor stem cell transplant technique shows promise

<p class="article-intro">In a new study, 90 % of young people with sickle cell disease (SCD) who received blood-forming stem cells from a parent were alive and healthy one year later, free of symptoms and complications. They did not need to take immune-suppressive medications, and in some instances their cognitive processing speed improved.</p> <hr /> <p class="article-content"><p>Currently, the only cure for SCD is a stem cell transplant using cells donated by a brother or sister who has the same tissue type and is SCD-free. According to lead study author Mitchell Cairo, MD, of New York Medical College in Valhalla, NY, only about one in six patients with SCD has a suitable sibling donor.</p> <p>The study presented at the ASH-congress focused on haploidentical transplants for SCD. In previous studies, however, the rate of transplant failure has been high. Cairo and his team sought to reduce both the rate of transplant failure and the risk of serious post-transplant complications by enriching CD34-positive stem cells. In addition, they added back the patients&rsquo; T-cells (after the transplant).</p> <p>In the study, 19 patients age 3&ndash;20 with frequent or severe SCD symptoms received transplanted stem cells; 15 patients received cells from their mothers, and 4 from their fathers.</p> <p>Engraftment occurred in all 19 patients in the study. At one year following the transplant, the rate of chimerism was 97 % . One patient developed acute GVHD within 100 days of the transplant and another developed chronic GVHD more than 100 days after the transplant.</p> <p>Follow-up tests performed two years after the transplant showed that patients had stable or improved heart and lung function. Imaging tests showed no evidence of strokes or inflammation of blood vessels in the brain, two potentially serious complications of SCD. Tests of memory, language, intellectual functioning, and ability to plan, focus on tasks, and manage emotions all showed stability or improvement.</p> <p><span lang="EN-US">These results suggest that 17 of 19 patients with SCD will go on to lead normal lives after undergoing a stem cell transplant that incorporates the techniques researchers used in this study, Dr. Cairo said. He cautioned, however, that the procedure is not successful in all patients, adding that whether patients will develop late adverse effects is not yet known.&nbsp;</span></p> <p><strong>Reference:</strong><br />Cairo M et al.: Significantly improved long term health related quality of life (HRQL) and neurocognition following familial haploidentical stem cell transplantation (HISCT) utilizing CD34 enrichment and mononuclear (CD3) addback in high risk patients with sickle cell disease (SCD). ASH Annual Meeting 2018, abstract #162</p></p>