Sickle Cell Disease and Hematopoietic Stem Cell Transplantation

This book provides a comprehensive, state-of-the art review of hematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD). The book reviews new data about risk prediction for severe SCD, outlines the unique challenges of HSCT for patients with SCD, profiles the supportive care guidelines for patients who are undergoing HSCT, highlights our current understanding of the best transfusion support for SCD patients prior to, during and after HSCT, and provides new perspectives about the ethics of HSCT for pediatric patients with SCD. Published in the last few years, several landmark phase III trials that utilize matched unrelated and haploidentical donors for HSCT in SCD patients are also placed in context with respect to current management.

Written by experts in the field, Sickle Cell Disease and Hematopoietic Stem Cell Transplantation is a valuable resource for physicians and researchers dealing with and interested in this challenging, yet exciting, curative therapy for sickle cell disease, that will help guide patient management and stimulate investigative efforts.

Outlines the unique challenges of HSCT and profiles supportive care guidelines for patients with SCD Reviews several landmark phase III trials that utilize matched unrelated and haploidentical donors for HSCT in SCD patients Written by experts in the field Includes supplementary material: sn.pub/extras

Autorentext
Emily Riehm Meier, MD, MSHS
Pediatric Hematologist and Director, Sickle Cell Research, Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA
Allistair Abraham, MDChildren's National Medical Center, Division of Blood and Marrow Transplantation, Washington, DC, USA
Ross M. Fasano, MDEmory University School of Medicine, Department of Pathology & Laboratory Medicine, Atlanta GA, USA

Inhalt
Clinical Manifestations of Sickle Cell Disease Across the Lifespan.- Pathobiology of Sickle Cell Disease Vaso-Occlusion and Targeted Therapies.- Current Non-HSCT Related Treatments for SCD.- Risk Based Therapies for Sickle Cell Disease.- Transfusion Support of the Patient with Sickle Cell Disease Undergoing Transplantation.- Neurological Manifestations of Sickle Cell Disease and Their Impact on Allogeneic Hematopoietic Stem Cell Transplantation.- Overview of Hematopoietic Stem Cell Transplantation for Non-Malignant Diseases.- Unique Challenges of Hematopoietic Stem Cell Transplant for Sickle Cell Disease.- The Ethics of Hematopoietic Stem Cell Transplantation for Sickle Cell Disease.- Psychosocial Care and Education of Children with Sickle Cell Disease undergoing Hematopoietic Stem Cell Transplant and their Families.- Long Term Effects of Hematopoietic Stem Cell Transplantation for Sickle Cell Disease.- Matched Sibling Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease.- Unrelated Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease.- Haploidentical Hematopoietic Cell Transplantation for Sickle Cell Disease.- Gene Therapy 101: The Path Toward Becoming a Realistic Cure for Patients with Sickle Cell Disease.