Hematopoietic stem cell transplantation: clinical use and perspectives

Abstract

Hematopoietic stem cell transplantation is the accepted therapy of choice for a variety of malignant and non-malignant diseases in children and adults. Initially developed as rescue therapy for a patient with cancer after high doses of chemotherapy and radiation as well as the correction of severe defi ciencies in the hematopoietic system, it has evolved into an adoptive immune therapy for malignancies and autoimmune disorders. The procedure has helped to obtain key information about the bone marrow environment, the biology of hematopoietic stem cells and histocompatibility. The development of this new discipline has allowed numerous groups working around the world to cure patients of diseases previously considered lethal. Together with the ever growing list of volunteer donors and umbilical cord blood banks, this has resulted in life saving therapy for thousands of patients yearly. We present an overview of the procedure from its cradle to the most novel applications, as well as the results of the HSC transplant program developed at our institution since 1989.

Bacterial bloodstream infections in the allogeneic hematopoietic cell transplant patient: new considerations for a persistent nemesis

“…16 In pediatric oncology patients, blood cultures drawn from the central CVC site that grow ⩾ 150 min before blood cultures from a peripheral site predict that the CLABSI is from a colonized CVC with 89% sensitivity and 100% specificity. 16,17 If peripheral blood cultures are not obtained, but blood cultures are obtained from both lumens of a double lumen CVC, 1 a differential time-to-positivity of ⩾ 180 min can be used to diagnose a CLABSI with 61% sensitivity and 94% specificity. However, this comparison has a poor negative predictive value.…”

“…1,2 Transplant strategies and supportive care have evolved over the past few decades, resulting in improved overall survival (OS). 3 Despite these advances, infection remains a primary cause of death following allo-HCT.…”

Cryopreservation of Hematopoietic Stem Cells: Emerging Assays, Cryoprotectant Agents, and Technology to Improve Outcomes

“…The major drawback of allogeneic HSCT is GVHD, which results in potentially very severe and life-threatening skin, gut, and liver disease. Allogeneic HSCT also may lead to delays in immune reconstitution, which can result in increased rates of infection, treatment-related mortality, and chronic GVHD [9-11]. Successful allogeneic HSCT also significantly relies on the availability of an appropriate donor source.…”

“…There are three major sources of hematopoietic stem cells (HSCs), including bone marrow harvested by aspiration from the cavity of the ilium (hipbone), peripheral blood obtained through leukapheresis, and umbilical cord blood (UCB) collected from the placenta after childbirth [9]. HSCT can be performed with either autologous HSCs (obtained from the patient) or allogenic HSCs (obtained from a donor), and both types of HSCs come with certain advantages and disadvantages.…”

Ruxolitinib for the Treatment of Patients with Steroid-Refractory GVHD: An Introduction to the REACH Trials

“…Allogeneic HSCT offers a potentially curative treatment option for several hematologic malignancies and disorders [1][2][3]. However, despite evolving prophylactic care in the field of transplantation, GVHD continues to pose a substantial burden and represents a leading barrier to HSCT success [3,18].…”