Cori Abikoff

Extracorporeal photopheresis practice patterns: An international survey by the ASFA ECP subcommittee

Although many apheresis centers offer extracorporeal photopheresis (ECP), little is known about current treatment practices.

Reduced Intensity Conditioning and Hematopoietic Stem Cell Transplantation in Pediatric Nonmalignant Disease: A New Therapeutic Paradigm

ematopoietic stem cell transplantation (HSCT) has shown promising advances during the past 50 years and is presently a curative option for several pediatric diseases. HSCT is often used as a treatment for recurrent or refractory malignancies, but more frequently is seen as an early therapeutic option in many nonmalignant diseases (NMDs). This is based on early successes such as severe combined immunodeficiency, where transplantation has an 80%-90% cure rate, and severe aplastic anemia, where HSCT has supplanted immunosuppressive therapy for patients with a matched sibling donor. Through direct replacement of cellular defects in the marrow or hematopoietic progenitor cells or the provision of circulating cells with normal enzymatic function to tissue where the enzyme is otherwise lacking, HSCT presents multiple attractive mechanisms to treat NMD. Historically, HSCT is preceded by a myeloablative conditioning (MAC) regimen, which leaves the recipient susceptible to deleterious side effects. Despite improvements in supportive care, there is a donor source–dependent, 10%27% treatment-related mortality (TRM) secondary to pediatric HSCT. Reported acute and chronic graft-versus-host disease (GVHD) rates are 56% and 33%, respectively. Particularly distressing for pediatric HSCT recipients are the late effects of MAC, which can lead to decades of chronic health-related problems. The National Cancer Institute summarized the late effects associated with pediatric HSCT recipients, including significantly greater rates of iron overload, chronic kidney disease, cardiac death, prolonged immunodeficiency, and endocrinopathies, including diabetes, growth impairment, osteopenia, and infertility. The risk of such complications limited the use of HSCT as a cure for NMDs, especially when the natural history was not one of rapid fatality, but shortened lifespan and significant morbidity. In diseases such as sickle cell disease