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Complications associated with allogeneic hematopoietic cell transplantation in children
1 Why is it important to understand complications after allogeneic hematopoietic cell transplantation? Allogeneic hematopoietic cell transplantation (AlloHCT) is a lengthy and complex process. The majority of the patients sustain complications after AlloHCT. These complications can have a significant impact…
Stem cell transplantation for children with nonmalignant disorders
The concept of stem cell transplant was first performed in 1956 in a patient with leukemia; however, this was quickly applied to nonmalignant disorders only a decade later in a patient with severe combined immunodeficiency. In patients with nonmalignant conditions,…
Hematopoietic cell transplantation for malignant disorders
1 How do the goals of autologous transplantation and allogeneic transplantation differ? Hematopoietic cell transplantation (HCT) is the act of restoring hematopoiesis by infusing hematopoietic stem cells. The cells that are infused can be from another person (allogeneic) or previously…
Gene therapy for hemoglobinopathies and other diseases
Hemoglobinopathies are genetically inherited conditions that originate from the lack or malfunction of components that comprise the hemoglobin (Hb) protein. Sickle cell disease (SCD) and thalassemia are the most common forms of these conditions. SCD is caused by a well-defined…
Car-T cells: expanding the universe of immunotherapy in children with malignant diseases
1 What are chimeric antigen receptor–T cells? Chimeric antigen receptor (CAR)-T cells are a powerful form of adoptive cell therapy. In their native state, T cells do not express antigens targeted to bind to malignant cells. In CAR-T therapy, T…
Autologous hematopoietic stem cell transplantation for children and young adults
Autologous hematopoietic stem cell transplant (auto HSCT) uses patient-derived stem cells to facilitate recovery from myeloablative high-dose chemotherapy for cancer. The concept is that higher doses of certain agents, particularly alkylators, will be 3 to 15 times more potent than…
Principles and practice of pediatric hematopoietic cell transplantation
1 Describe the types of transplant and their indications. Type of Transplant Details of Transplant Indications Disadvantages Autologous hematopoietic stem cell transplantation (autoHCT) Use patient’s own hematopoietic cells. Give granulocyte colony-stimulating factor (G-CSF) and/or Plerixafor to mobilize cells to collect.…
Hemophagocytic lymphohistiocytosis
1 What is the definition of hemophagocytic lymphohistiocytosis? Hemophagocytic lymphohistiocytosis (HLH) is a rare but potentially fatal clinical syndrome of severe pathologic immune activation, characterized by signs and symptoms of excessive inflammation. It commonly appears in infancy but can be…
Langerhans Cell Histiocytosis (LCH)
1 What are the former names of Langerhans cell histiocytosis? What is known today as Langerhans cell histiocytosis (LCH) was first described in the early 1900s. By 1953, the diagnoses of eosinophilic granuloma, Hand-Schüller-Christian disease, Letterer-Siwe disease, and Hashimoto-Pritzker disease…