Hematopoietic stem cell transplantation (HSCT) is an important treatment option for children with severe and refractory sickle cell disease (SCD) with debilitating clinical complications. HSCT with cells from the bone marrow of a HLA-identical sibling used in SCD has a low mortality risk, high cure rate, and high event-free survival rate after a median follow-up of 5-6 years. However, matched donors are found in only about 20% of the patients. A boy aged 8 years with SCD had a sister, <2 years old, a fully compatible donor. The boy met all eligibility criteria to undergo HSCT, and he was suffering from cognitive and neurologic impairment due to ischemic events. A Bioethical Committee jointly discussed the ethical issues on this case after a pediatric evaluation released the very young sister for donation. The justification was that the sister would benefit from the donation too because of the greater likelihood of survival and cure and less suffering of her brother. The parents were informed about the risks and benefits for both children, and the family was psychologically evaluated. After their consent, HSCT was performed and the patient is cured from SCD. The complication for the donor was the need for blood transfusion.
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http://dx.doi.org/10.1155/2017/8394732 | DOI Listing |
Transpl Infect Dis
December 2024
Department of Infectious Diseases and Immunology, Austin Health, Heidelberg, Australia.
Background: Identifying patients with latent tuberculosis infection (LTBI) is challenging. This is particularly true amongst immunocompromised hosts, in whom the diagnostic accuracy of available tests is limited. The authors evaluated the impact of routine pretransplant review by a transplant infectious diseases (TID) physician on LTBI screening in allogeneic hematopoietic stem cell transplant (alloHSCT) recipients.
View Article and Find Full Text PDFAnn Med
December 2025
Department of Hematology, Affiliated Hangzhou First People's Hospital, Westlake University, School of Medicine, Hangzhou, China.
Background: The management of high-risk acute myeloid leukaemia (AML) remains challenging, highlighting the need for innovative conditioning strategies beyond current regimens.
Methods: In the present single-arm study, a FACT regimen comprised of low-dose total body irradiation (TBI) with fludarabine, cytarabine and cyclophosphamide was employed to treat cytogenetically high-risk AML patients exhibiting pre-transplant active disease. This clinical trial is registered in the Chinese Clinical Trial Registry with the registration number ChiCTR2000035111.
Sci Rep
December 2024
Faculty of Medicine, University of Social Sciences in Łódź, 90-113, Łódź, Poland.
Due to its course, multiple myeloma may negatively affect the functioning of patients. Different treatment methods are also associated with patients' varying perception of their health condition. The purpose of this study is to determine the disease-specific complaints among multiple myeloma patients during selected treatment methods-chemotherapy, autologous hematopoietic stem cell transplantation, and supportive therapy.
View Article and Find Full Text PDFSci Rep
December 2024
Clinic of Neurology and Neurosurgery, Institute of Clinical Medicine, Faculty of Medicine, Vilnius University, Vilnius, Lithuania.
Immune reconstitution therapy (IRT) is a relatively new and highly effective treatment option for multiple sclerosis (MS). Uncertainty regarding the development of autoimmune disorders (ADs) after some therapies remains. The aim of this study was to assess new AD development after IRT in MS patients and to describe the nature of those ADs and the time to onset.
View Article and Find Full Text PDFMol Ther
December 2024
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita - Salute San Raffaele University Medical School, Milan, Italy; Pediatric Immunohematology and BMT, San Raffaele Hospital, Milan, Italy.
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