Gene Therapy for Primary Immunodeficiencies
The concept of gene therapy emerged as a way of correcting monogenic inherited diseases by introducing a normal copy of the mutated gene into at least some of the patients’ cells. Although…
Hematopoietic Stem Cell Transplantation: An Overview of Infection Risks and Epidemiology
Hematopoietic stem cell transplantation (HSCT) is a treatment for multiple medical conditions that result in bone marrow failure and as an antineoplastic adoptive immunotherapy for hematologic malignancies. HSCT is associated…
Purified Hematopoietic Stem Cell Transplantation: The Next Generation of Blood and Immune Replacement
Replacement of disease-causing stem cells with healthy ones has been achieved clinically via hematopoietic cell transplantation (HCT) for the last 40 years, as a treatment modality for a variety of…
Haploidentical Bone Marrow Transplantation in Primary Immune Deficiency: Stem Cell Selection and Manipulation
Since the early 1980s T-cell depletion has allowed haploidentical bone marrow transplantation to be performed in patients with primary immunodeficiency for whom a matched sibling donor was not available, without…
HLA-haploidentical Donor Transplantation in Severe Combined Immunodeficiency
Curative treatment of Severe Combined Immunodeficiency (SCID) by Hematopoietic Cell Transplantation (HCT) remains a challenge, in particular in infants presenting with serious, poorly controllable complications. In the absence of a…
A History of Bone Marrow Transplantation
The last 40 years has seen the emergence of hematopoietic stem cell transplantation as a therapeutic modality for fatal diseases and as a curative option for individuals born with inherited…
Hematopoietic Stem Cell Transplantation for Severe Combined Immune Deficiency or What the Children have Taught Us
It is now more than 40 years since the first successful allogeneic hematopoietic stem cell transplantation (HSCT) for a child with severe combined immunodeficiency (SCID). In the succeeding years, HSCT…
Anesthesia in the Pregnant Patient with Hematologic Disorders
The management of patients with hematologic disorders in pregnancy involves a multidisciplinary approach involving specialists from hematology, obstetrics, and anesthesiology. Whereas regional anesthesia has led to a decline in maternal…
Hematologic Disease in Pregnancy: The Obstetrician’s Perspective
Pregnancy can be a time of significantly increased morbidity and mortality in women with hematologic disease. With careful planning and preparation, most women can be cared for safely, resulting in…
Factor Deficiencies in Pregnancy
Pregnancy, childbirth, and the puerperium are hemostatically challenging to women with bleeding disorders. This article provides general recommendations for the management of pregnant women with inherited coagulation disorders. Each factor…
