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Hematopoietic cell transplantation (HCT) transfers a small number of hematopoietic stem cells (HSC) from a donor to a recipient, where they are able to differentiate and proliferate to restore a normal hematopoietic and immune system (see eFig. 133.1). HCT is performed in patients with life-threatening hematologic disorders or as a means to restore hematopoietic function following administration of otherwise lethal doses of chemotherapy or radiation for treatment of resistant malignancies. HCT also has the potential to cure disorders resulting from defects in the pluripotent progenitor cells or in a single hematopoietic lineage.

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eFigure 133.1.
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Development of hematopoietic cells from the pleuripotent stem cell. Characteristics of the hematopoietic stem cell include the capacities for differentiation and for self-renewal. BFU, burst forming unit; CFU, colony forming unit; CFU-GEMM, Colony-forming unit—granulocyte, erythrocyte, monocyte, macrophage; E, erythroid; Meg, megakaryocyte; GM, granulocyte/macrophage; Eo, eosinophil.

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HCT is most commonly used to treat aggressive hematopoietic malignancies that have not responded to conventional therapy (see eFig. 133.2). Acute lymphoblastic leukemia is the most common indication for HCT in children who fail to achieve remission following induction chemotherapy, or in those that relapse following chemotherapy. In general, 65% to 75% of patients in first remission, 40% to 50% of those in second remission, and 10% of those with more advanced disease will survive long term (see Chapter 449).1,2HCT is not typically performed in first remission unless the leukemia has features such as the Philadelphia chromosome, which predicts a poor prognosis with conventional chemotherapy. In patients with acute myeloid leukemia, HCT is generally indicated at first remission if an HLA–identical sibling donor is available (see Chapter 450).3 Other hematologic malignancies, such as chronic myelogenous leukemia, juvenile chronic myeloid leukemia, myelodysplastic syndromes, and myeloproliferative syndromes, are considered appropriate candidates for HCT, and in certain instances, HCT is the only potential modality for cure.4,5

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eFigure 133.2.
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Proportion of pediatric patients given hematopoietic cell transplant according to diagnosis (left panel) or donor cell source (right panel).

ALL, Acute lymphoblastic leukemia; AML, Acute myelogenous leukemia; CML, Chronic myelogenous leukemia; MDS, myelodysplastic syndromes; MRD, HLA-matched related donor; Auto, Autologous; Alternative, Alternative stem cell donors.

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Hematopoietic cell transplantation (HCT) is also used to treat some pediatric solid tumors. The hematopoietic cells generally come from the patient, although other donors have been used. The transplant typically is performed after chemotherapy, and sometimes surgery or radiation, has reduced the tumor burden sufficiently. Neuroblastoma is the most common solid tumor for which HCT is used (see Chapter 457). Results of randomized studies have indicated that HCT, when performed at a point of minimal disease, improves survival.6 Patients with relapsed Hodgkins or non-Hodgkins lymphoma also may benefit from HCT, which results in approximately 50% long-term survival when ...

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