Hemopoietic Stem Cell Transplant for Non-Hematological Disorders

Abstract

This chapter will consider and summarize Hematopoietic Cell Transplant (HCT) in metabolic disorder and in osteopetrosis. The role of HCT in these disorders is perhaps less obvious since the blood cells themselves function normally and there is no malignancy. The application of HCT in non-hematological disease is a growing area and is likely to concern all transplant physicians in the coming decades.

HCT in leukemia is intuitive, even to the lay person. There is a bone marrow malignancy, and so change that marrow for a healthy one. Similarly, in aplasia the transplant corrects hematopoietic stem cell number and function, and in the hemoglobinopathies and primary immune deficiencies, the mature progeny of the transplanted, healthy donor cells are functional, and disease is corrected.

In this chapter, some of those illnesses in which HCT is less intuitive are discussed. In these illnesses the HCT might attenuate illnesses and improve patients despite their being nothing wrong with the blood itself in the first place. The blood might be the best part of some of these patients, and yet to correct disease manifestations then all that is corrected is this blood.

The use of HCT in inborn errors of metabolism (IEM) is discussed, including in the lysosomal storage diseases (LSD), X-linked adrenoleukodystrophy (X-ALD), and its use in malignant infantile osteopetrosis (MIOP). The scope of transplant in this type of illness is considered, and how it may change in future years is signposted. Both the generic principles of transplant in metabolic diseases and the specifics of the management of the commoner indications will be covered.