https://ebmt.co.uk/wp-content/uploads/2013/08/GVHD1.pdf

National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: I. Diagnosis and Staging Working Group Report

Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel

BACKGROUND: The cause of chronic myeloid leukemia (CML) is a constitutively active BCR-ABL tyrosine kinase. Imatinib inhibits this kinase, and in a short-term study was superior to interferon alfa ...

/pdf/five-year-follow-up-of-patients-receiving-imatinib-for-2ejt1h30j2.pdf

Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia

http://caprockhematology.com/Site/AML_and_ALL_files/AML%20Review%20Blood%202010.pdf

Diagnosis and management of acute myeloid leukemia in adults: recommendations from an international expert panel, on behalf of the European LeukemiaNet.

Publisher Summary Studies of cytotoxicity by human lymphocytes revealed not only that both allogeneic and syngeneic tumor cells were lysed in a non-MHC-restricted fashion, but also that lymphocytes from normal donors were often cytotoxic. Lymphocytes from any healthy donor, as well as peripheral blood and spleen lymphocytes from several experimental animals, in the absence of known or deliberate sensitization, were found to be spontaneously cytotoxic in vitro for some normal fresh cells, most cultured cell lines, immature hematopoietic cells, and tumor cells. This type of nonadaptive, non-MHC-restricted cellmediated cytotoxicity was defined as “natural” cytotoxicity, and the effector cells mediating natural cytotoxicity were functionally defined as natural killer (NK) cells. The existence of NK cells has prompted a reinterpretation of both the studies of specific cytotoxicity against spontaneous human tumors and the theory of immune surveillance, at least in its most restrictive interpretation. Unlike cytotoxic T cells, NK cells cannot be demonstrated to have clonally distributed specificity, restriction for MHC products at the target cell surface, or immunological memory. NK cells cannot yet be formally assigned to a single lineage based on the definitive identification of a stem cell, a distinct anatomical location of maturation, or unique genotypic rearrangements.

Biology of natural killer cells.

Fanconi anemia (FA) is the most frequent inherited cause of bone marrow failure. Most FA patients experience hematopoietic stem cell attrition and cytopenia during childhood. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for FA patients in case of bone marrow failure or clonal evolution (i.e., myelodysplastic syndrome or acute myeloid leukemia). HLA identical HSCT is still the best treatment but drastic improvement has been shown using an unrelated donor. Alternative transplantation (i.e., cord blood-, mismatch-, or haplo-HSCT) is still experimental and should not be done outside experienced centers. FA diagnosis, optimal HSCT timing, best conditioning regimen, as well as, long-term follow-up of those patients are addressed in this paper.

Chapter 16 – Fanconi Anemia

Outcomes of graft failure after umbilical cord blood transplantation in acute leukemia: a study from Eurocord and the Acute Leukemia Working Party of the EBMT

Umbilical cord blood (UCB) stem cells [4] are hematopoietic stem cells [5] (HSC) that are recovered from the blood of the umbilical cord [6] and placenta [7] after birth. Umbilical cord blood is rich in cells that express the CD34 molecule, a surface protein that identifies cells as stem cells [4] . Prior to the discovery of UCB stem cells [4] , it was standard procedure to discard the umbilical cord [6] and placenta [7] ; now much effort is devoted to raising public awareness and to encouraging people to store or donate cord blood. The importance of these cells lies in potential clinical treatments of blood-borne diseases, as well as the possibility of restoring cells of other lineages, such as cardiac and neural cells. These possible uses have given rise to cord blood stem cell banking [8] , both private and public, where cells can be frozen and stored for later use. their Umbilical Cord Blood as a Potential Source of Transplantable Hematopoietic Stem/Progenitor Cells,” . cord and placenta; now much effort is devoted to raising public awareness and to encouraging people to store or donate cord blood. The importance of these cells lies in potential clinical treatments of blood-borne diseases, as well as the possibility of restoring cells of other lineages, such as cardiac and neural cells. These possible uses have given rise to cord blood stem cell banking, both private and public, where cells can be frozen and stored for later use.

Umbilical Cord Blood Stem Cells (UCBSC) [1]

EBV transformation of cells from cord blood donations: relevance to future retrospective studies on cord blood transplants

EBV transformation of cells from cord blood donations: relevance to future retrospective studies on cord blood transplants.

Aplastic anaemia can be cured either by bone marrow transplantation, which leads to stem cell replacement after a myeloablative conditioning regimen, or with nonreplacement therapy, often referred to as immunosuppressive therapy. Antilymphocyte globulin and cyclosporin are used, either alone or in association, as the cornerstone of immunosuppressive therapy for aplastic anaemia. Both cyclosporin and antilymphocyte globulin used alone induce remission in nearly 50% of patients, and long term follow-up of large cohorts indicates survival rates in the range of 60 to 70%. Immunosuppressive therapy should be first-line treatment for patients without a tissue-identical sibling and for those over 40 to 45 years of age. However, aplastic anaemia is a rare disease and some questions regarding the optimum immunosuppressive therapy remain to be answered. As a rule in rare diseases, these questions are best answered through multicentre prospective randomised trials, now in progress.

https://link.springer.com/content/pdf/10.1007%2FBF03259068.pdf

Eliane Gluckman

Papers

Chapter 16 – Fanconi Anemia

Outcomes of graft failure after umbilical cord blood transplantation in acute leukemia: a study from Eurocord and the Acute Leukemia Working Party of the EBMT

Umbilical Cord Blood Stem Cells (UCBSC) [1]

EBV transformation of cells from cord blood donations: relevance to future retrospective studies on cord blood transplants.

Acquired Aplastic Anaemia