Sickle Cell Disease and Hematopoietic Stem Cell Transplantation

1. Front Matter

   Pages i-xiii

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2. Sickle Cell Disease

   1. Front Matter

      Pages 1-1

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   2. Clinical Manifestations of Sickle Cell Disease Across the Lifespan

      • Lydia H. Pecker, Jane Little

      Pages 3-39

   3. Pathobiology of Sickle Cell Disease Vaso-occlusion and Targeted Therapies

      • Kerry A. Morrone, Jennifer Davila, W. Beau Mitchell, Deepa Manwani

      Pages 41-64

   4. Current Non-HSCT Treatments for SCD

      • Claire L. Anderson, Deepika S. Darbari

      Pages 65-86

   5. Risk-Based Therapies for Sickle Cell Disease

      • Emily Riehm Meier

      Pages 87-110

   6. Transfusion Support of the Patient with Sickle Cell Disease Undergoing Transplantation

      • Jeanne E. Hendrickson, Ross M. Fasano

      Pages 111-136

   7. Neurological Manifestations of Sickle Cell Disease and Their Impact on Allogeneic Hematopoietic Stem Cell Transplantation

      • Jessica L. Carpenter, Monica L. Hulbert

      Pages 137-159

3. Hematopoietic Stem Cell Transplantation

   1. Front Matter

      Pages 161-161

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   2. Overview of Hematopoietic Stem Cell Transplantation for Nonmalignant Diseases

      • Karen L. Zimowski, Shanmuganathan Chandrakasan

      Pages 163-184

   3. Unique Challenges of Hematopoietic Stem Cell Transplant for Sickle Cell Disease

      • Pooja Khandelwal, Michael Grimley

      Pages 185-198

   4. The Ethics of Hematopoietic Stem Cell Transplantation for Sickle Cell Disease

      • Robert Sheppard Nickel, Naynesh Kamani

      Pages 199-219

   5. Psychosocial Care and Education of Children with Sickle Cell Disease Undergoing Hematopoietic Stem Cell Transplant and Their Families

      • Steven J. Hardy, Jasmine R. Holt, Amanda L. Thompson

      Pages 221-233

   6. Long-Term Effects of Hematopoietic Stem Cell Transplantation for Sickle Cell Disease

      • Karim T. Sadak, Angela R. Smith, Monica Bhatia

      Pages 235-249

   7. Matched Sibling Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease

      • Gregory M. T. Guilcher, John T. Horan

      Pages 251-269

   8. Unrelated Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease

      • Alexander Ngwube, Shalini Shenoy

      Pages 271-281

   9. Haploidentical Hematopoietic Cell Transplantation for Sickle Cell Disease

      • Elizabeth O. Stenger, Allistair Abraham

      Pages 283-302

   10. Gene Therapy: The Path Toward Becoming a Realistic Cure for Sickle Cell Disease

       • Alexis Leonard, Allistair Abraham

       Pages 303-328

4. Back Matter

   Pages 329-334

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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.

• hydroxyurea
• molecular phenotyping
• silent cerebral infarcts
• reduced intensity conditioning
• transfusional hemochromatosis
• GVHD prophylaxis

“An authoritative work on hematopoietic stem cell transplantation (HSCT) … . Written by leading expert clinicians and researchers … . this is a very valuable resource for clinicians and scientists dealing with this devastating disease and pursuing a curative therapy.” (Ravi Salgia, Doody's Book Reviews, August, 2018)

• Indiana Hemophilia and Thrombosis Center, Indianapolis, USA

  Emily Riehm Meier

• Division of Blood and Marrow Transplantation, Children’s National Medical Center, Washington, DC, USA

  Allistair Abraham

• Department of Pathology and Laboratory Medicine, Emory University School of Medicine, Atlanta, USA

  Ross M. Fasano

Emily Riehm Meier, MD, MSHS

Pediatric Hematologist and Director, Sickle Cell Research, Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA


Allistair Abraham, MD
Children’s National Medical Center, Division of Blood and Marrow Transplantation, Washington, DC, USA


Ross M. Fasano, MD
Emory University School of Medicine, Department of Pathology & Laboratory Medicine, Atlanta GA, USA

Policies and ethics