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Bone marrow transplantation.

Bone marrow transplantation can be considered in any disease state resulting in the malfunction or absence of part or all bone marrow elements. Diseases such as aplastic anemia, leukemia, and immunodeficiency disease are being treated with bone marrow transplantation. As with any organ transplant, graft rejection is a possibility. In bone marrow transplantation, there is the additional, unique problem of graft versus host disease. In order to prevent or minimize graft rejection, the immunocompetence of the recipient and the degree of disparity between donor and recipient at the major histocompatibility complex (MHC) loci are considered. The results of bone marrow transplantation are variable, and the mortality rate is still relatively high. However, progress is being made, and in many instances, normal bone marrow function can be restored in patients with whom other treatment has failed.

Anemia, Aplastic

Stages in pediatric bone marrow transplantation.

Bone marrow transplantation is a relatively new and aggressive procedure that is being used in the treatment of leukemia and aplastic anemia. We observed approximately 30 children and their families at the UCLA Medical Center throughout the procedure. Various psychological responses during the treatment are outlined. Eleven stages are identified, including the patient's and family's first awareness of this procedure, the preadmission evaluation and psychosocial assessment, the introduction into isolation, the donor's hospitalization, the transplantation itself, and the various reactions and interactions of the patient, family, and staff throughout these stages.

Anemia, Aplastic

Bone marrow transplantation from donors with aplastic anemia. A report from the ACS/NIH bone marrow transplant registry.

Bone marrow transplantation from HLA-matched allogeneic donors was used to treat two series of patients with severe aplastic anemia. No significant differences were detected in all comparisons made between the Registry series (38 patients) and the Seattle series (24 patients), and pooled data from the two series were used in the analyses. Currently, 55% of the patients who received bone marrow transplants within three months of diagnosis are alive, but only 13% of the patients who received bone marrow transplants more than nine months after diagnosis are alive. The difference between the two groups was significant (P less than .02). Patients less than 21 years of age had a significantly higher survival rate than those patients who were 21 years or older at the time of transplantation (P less than .02). Survival rates were significantly higher for patients who had received 15 or fewer pretransplant transfusions than those who received more (P less than .05).

Adolescent

[Attempt to treat a case of chronic familial granulomatous disease by allogenic bone marrow transplantation].

A bone marrow graft was performed in a 3 1/2 year old boy suffering from chronic granulomatous disease. Donor-recipient matching was complete in the ABO, HL-A and M.L.C. systems. The number of injected cells amounted to 5.4 X 10(9) (3.6 X 10(8) cells/kg body weight). The immunosuppression regimen consisted of cyclophosphamide and A.L.S. before the graft, and methotrexate after it. Neither graft versus host reaction, nor secondary infection developed. The take of the graft was monitored by the Gm allotypes. Rejection, however, occurred after 2 months.

Adolescent

A solution to the graft-versus-host problem in bone marrow transplantation to humans.

Many clinical situations arise where it would be desirable to transplant bone marrow to a marrow function deficient patient. However, bone marrow is immunologically competent by virtue of its content of lymphocyte precursors. Marrow transplantation in these patients is followed by the graft's immunological rejection of the patient - a fatal disease. A system for specific abrogation of this graft-versus-host disease after xenogeneic bone marrow transplantation to humans is presented. In this system, prospective patient cells are removed by skin biopsy for example, and injected into a fetal chimpanzee. The fetal chimpanzee becomes tolerant to this patient's antigens and will not attack or reject them when its bone marrow is removed after birth and injected into the specific patient from whom the tolerogenic cells were obtained. A simple and straightforward experimental test of this system's clinical applicability is also presented.

Animals

Bone marrow transplantation in man.

Bone marrow transplantation is emerging as a viable therapeutic approach to a number of diseases that are usually or uniformly fatal. We review here recent experiences in bone marrow transplantation in man at UCLA and in various other institutions throughout the world. We examine marrow transplantation in immunodeficiency diseases, acute leukemia, and aplastic anemia and consider the problems of infection in the transplant recipients. The applications of tissue typing to marrow transplantation and immunologic manipulations, which may influence engraftment and graft-versus-host disease, are also reported.

ABO Blood-Group System

Bone marrow transplantation in children.

Bone marrow transplantation is a new concept in the treatment of leukemia and aplastic anemia. The problems seen in the transplanted patient are abundant and often life-threatening. Caring for these patients offers one of the most exciting challenges of nursing. The nurse not only acquires skill in caring for a patient with leukemia and aplastic anemia, but also becomes knowledgeable in infectious diseases, immunology, fluid and electrolyte balance, and cardiac, respiratory, and renal diseases. The complexity of these patients allows the nurse more direct, comsistent contact with them. As she becomes involved, she is given excellent opportunities for supporting the patient and family through this stressful time. Althoug bone marrow transplantation is an experimental procedure with problems still to be solved, results indicate its value in the treatment of refractory leukemia and aplastic anemia.

Anemia, Aplastic

Morphologic aspects of bone marrow transplantation in patients with aplastic anemia.

Pre- and post-transplant bone marrow samples from 20 patients with aplastic anemia were studied. Morphologic evidence of marrow reconstitution was noted in 18 patients one to three weeks following transplantation. In most instances the engrafted marrow elements in early weeks appeared as small clusters of erythroid or myeloid precursors. Bone marrow biopsy or clot sections obtained four to eight weeks after transplantation were more cellular with larger clusters of hematopoietic cells, which were most often composed of mixed cellular elements, including megakaryocytes. Two patients with morphologic evidence of engraftment died shortly after transplantation and were excluded from further analysis. In four of the remaining 16 patients grafts were "rejected" three to eight weeks after transplant. A fifth patient who received a marrow graft from his identical twin showed a transient increase in marrow cellularity without clinical improvement. However, the second marrow transplantation in this patient using the same donor after conditioning with cyclophosphamide resulted in moderate clinical improvement. In four of the five patients developing graft "rejection" or failure there was an increase in marrow mast cells in pre- and post-transplant marrow samples. In contrast, only two of 11 patients with successful engraftment had an increase in mast cells. Although the pathophysiologic role of mast cells in marrow transplantation is unclear, the present study suggests a possible inverse correlation between the numbers of marrow mast cells and the likelihood of successful engraftment. Bone marrow samples in patients with graft versus host disease displayed a slight increase in the number of eosinophils, lymphocytes, and plasma cells.

Adolescent

Obstructive jaundice after bone marrow transplantation.

Jaundice after bone marrow transplantation is usually a consequence of graft versus host disease. Reported is a patient who presented with obstructive jaundice several months after a successful marrow allograft. Despite a benign bone marrow examination, abdominal ultrasound, upper gastrointestinal series, and endoscopic biopsy were utilized to diagnose recurrent leukemia at the pancreatic head and descending duodenum. The entities of graft versus host disease as related to jaundice, and gastrointestinal leukemia, in the presence of a "remission" bone marrow, are reviewed.

Biopsy

Chediak-Higashi syndrome: reversal of increased susceptibility to infection by bone marrow transplantation.

Transplantation of normal bone marrow to mice with the Chediak-Higashi syndrome (CHS) resulted in normal granulopoiesis and a reversal of their increased susceptibility to challenge with intravenous Candida albicans. These findings suggest that (1) the leukocyte defect in CHS can be reversed by marrow transplantation and (2) the mechanism for increased susceptibility to infection in these animals is due to a bone-marrow-derived cellular defect. Because of similarities between murine and human CHS, bone marrow transplantation might be considered as a mode of therapy in selected cases of the human disease.

Animals