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PubMed2002

Myelodysplastic syndrome complicated by autoimmune hemolytic anemia: remission of refractory anemia following mycophenolate mofetil.

Autoimmune hemolytic anemia (AIHA) rarely occurs in myelodysplastic syndrome (MDS). A 36-year-old Asian female was diagnosed with MDS (refractory cytopenia with multilineage dysplasia, RCMD) and complicated by AIHA 7 months later. Secondary myelofibrosis developed at the same time. Steroid therapy was ineffective and cyclosporin A (CsA) was discontinued due to its neurotoxicity with the development of leukoencephalopathy. However, the patient achieved a good hematological response after the use of mycophenolate mofetil (MMF, CellCept) with a dose of 1 g/day and prednisolone (15 mg/day). Prednisolone was tapered off over the next 3 weeks. The patient did not require any blood support 4 weeks after the use of MMF and has been hematologically stable for 4 months. To our knowledge, this is the first report of using MMF in treating MDS complicated by AIHA. MMF might be considered as a salvage therapy for patients with refractory anemia complicated by AIHA.

Adult↗
PubMed1976

Ultrastructure of platelet aggregation in refractory anemia and myelomonocytic leukemia. I. Ultrastructure of aggregation in normal controls and general defects in refractory anemia and myelomonocytic leukemia.

In vitro aggregation of the platelets from four patients with refractory anemia and two patients with acute myelomonocyctic leukemia revealed distinctive abnormalities. In five patients, there was deficient or minimal aggregation with adenosine diphosphate (ADP), epinephrine, or collagen and only one wave of aggregation could be elicited with ADP at any concentration. Ultrastructural studies revealed numerous isolated platelets, small aggregates with few platelet pseudopods, and the presence of a characteristic type of aggregate with heterogeneous platelet composition combining features of both the primary and the secondary waves of aggregation. These "mixed aggregates" were particularly abundant in the four patients who had refractory anemia and may constitute the structural basis of the single wave of aggregation observed.

Adenosine Diphosphate↗
PubMed2002

Morbidity and mortality of chronic GVHD after hematopoietic stem cell transplantation from HLA-identical siblings for patients with aplastic or refractory anemias.

We analyzed effects of successive changes in prevention and treatment of chronic GVHD in 405 patients with aplastic anemia and refractory anemia given HLA-matched hematopoietic stem cell transplantation (HSCT) from 1970-1997. For analysis, patients were divided into group I, transplantations from 1970-1976; group II, 1977-1983; group III, 1984-1990; and group IV, 1991-1997. The overall incidence of chronic GVHD was 28%. Incidences of chronic GVHD for groups I through IV were 20%, 46%, 41%, and 22%, respectively, reflecting added buffy coat infusions in groups II and III. Five-year survival rates of patients with chronic GVHD for groups I through IV were 58%, 74%, 82%, and 76%, respectively (NS). Among group I patients, 50% were alive off immunosuppression, none were alive on immunosuppression, and 50% died. These figures were 76%, 0%, and 24% in group II; 80%, 10%, and 10% in group III; and 64%, 21%, and 14% in group IV patients. More serious infections and skin contractures were seen in group I than in groups II, III, and IV (P = .0001, .02, .01 and P =.0003, .001, .05, respectively). Lung complications, aseptic necroses, depression, and Karnofsky scores were comparable among groups. Gastrointestinal complications seemed less frequent among groups II through IV. Diabetes mellitus was more frequent in group IV than in groups I through III (P = .008). Secondary malignancies occurred in 33%, 6%, 3%, and 0% of patients in the 4 groups, respectively. In conclusion, over 28 years, chronic GVHD has remained challenging, with only slight improvements in quality of life. Decisive improvements in therapy and survival will have to await both a better understanding of the immunological events underlying chronic GVHD and better infection prevention and control.

Adolescent↗
PubMed1979

Refractory anemia with excess of blast cells: prognostic factors and effect of treatment with androgens or cytosine arabinoside. Results of a prospective trial in 58 patients. Cooperative Group for the Study of Aplastic and Refractory Anemias.

The results of a prospective study of 58 patients with refractory anemia and partial blastic infiltration of the bone marrow lead to the following conclusions. The median survival (12 months from diagnosis) is shorter and the rate of acute leukemia as cause of death (60%) higher than in other retrospective series. This group of patients, however, appears to be a "continuum" of preleukemic states with more or less rapid evolution, so that the exclusion of the most severe cases appears unjustified. Based on the degree of bone marrow blastosis, and also on the degree of blood cytopenias, the anomalies of 59 Fe incorporation kinetics and the bone marrow stem-cell cultures, it is possible to derive a plausible prognosis for individual patients, which could aid the choice of therapy. Androgen therapy does not accelerate leukemic evolution, but does not improve the bone marrow insufficiency. Cytosine-arabinoside at low dosage exhibited no toxicity, but did not delay the appearance of overt leukemia.

Acute Disease↗
PubMed1978

Association of 5q- and refractory anemia.

The association of refractory anemia with partial deletion of the long arm of one No. 5 chromosome (5q-) has been reported in six patients within the last two years. We wish to report an additional patient with refractory anemia and an acquired chromosomal deletion, del(5)(q15), in a direct bone-moarrow preparation. The specificity of this deletion for refractory anemia is discussed. It remains to be seen whether patients with refractory anemia and 5q- tend to develop acute leukemia, as has been recently suggested.

Acute Disease↗
PubMed1980

Studies on the pathogenesis of refractory anemia.

Nine patients with refractory anemia were studied using the soft agar marrow culture assay (CFU-c) to identify granulocyte-monocyte progenitor cells. Patients' marrows were then cocultured with normal marrow to identify suppressor cells that inhibit normal colony formation. Three of nine patients had low colony formation and no suppression in coculture. These patients may have a defect intrinsic to the marrow granulocyte-monocyte progenitor cell, termed type I. Three of nine patients had normal colony formation and no suppression in coculture, possibly representing a type II defect in the hemopoietic environment. Three of nine patients had low colony formation in the CFU-c assay and their marrow contained cells that suppressed colony formation by normal marrow in coculture. This defect, termed type III, may result from suppressor cells. Thus, refractory anemia may be a syndrome resulting from at least three different pathogenetic mechanisms involving defects in (1) stem cells, (2) the marrow environment or (3) suppressor cells. This may represent one end of the spectrum of pancytopenia with diminished cellularity (aplastic anemia) or normal cellularity (refractory anemia) resulting from similar mechanisms.

Adult↗
PubMed2000

Successful allogeneic bone marrow transplantation for childhood-onset refractory anemia with ringed sideroblasts.

Refractory anemia with ringed sideroblasts (RARS) is an extremely rare type of myelodysplastic syndrome in children. We describe a 10-year-old boy with RARS presented with pancytopenia. He remained relatively stable with only a few transfusions until age of 20 years, when he underwent an allogeneic bone marrow transplantation (BMT) because of increased transfusion requirements. He remains in complete chimeric state at 20 months posttransplant with normal hematologic parameters. To our knowledge, this is the first description of successful BMT in a patient with childhood-onset RARS. The indication of BMT for this rare disorder in children is discussed.

Anemia, Refractory↗
PubMed1986

Refractory anemia.

Explore the source record for details and available documents.

Anemia, Refractory↗
PubMed1990

[A case of refractory anemia with excess of blasts (RAEB) having weakened expression of B blood group antigen].

A 52 year-old male was admitted to our hospital with the complaint of the hypochondrial pain after meal in February 1989. He was diagnosed to be RAEB and to have common bile duct stone. His red blood cells (RBC) could not be agglutinated with anti-A, anti-B, or anti-A, B. The agglutinability of the cells to Ulex europaeus (anti-H) was the same to the normal type B RBC, and his serum contained anti-I, anti-E, and anti-c. In his saliva, both B and H antigens were detected. The glycosyl B transferase in his serum showed similar activity to that of normal individual with type B RBC. When his RBC were treated with normal type B transferase, the cells obtained the reactivity to anti-B but they agglutinated much weaker than the type O cells treated with the enzyme. These results indicated that the low agglutinability of his RBC could not be due to the low transferase activity but the qualitative or quantitative changes of the precursor molecules of the type B substance of the cells.

ABO Blood-Group System↗
PubMed1988

[Therapy of the preleukemic state: effect of androgens on refractory anemia].

We have examined the efficacy of various drugs in 44 patients with MDS and found the different effectiveness which depends on the type of MDS. Namely, RA appears to respond to steroid hormone, androgen, and/or vitamin D3, regardless of single or combined use. In particular, it is obvious in androgen, and as our previous reports, high content of acidic ferritin in RBC with RA have changed to more basic ones by treatment with androgen. On the contrary, these drugs were not effective on RAEB, RAEB-T, and CMML. A long-term observation is needed to determine whether the prolonged or decreased occurrence of leukemia could be obtained in the effective cases with RA. Most of the cases who did not develop overt leukemia during this study died of bleeding or infections due to thrombocytopenia or leukocytopenia, thus indicating that supportive therapies are important in patients with MDS. Since it has recently been reported that recombinant G-CSF or GM-CSF is helpful to increase the number of leucocyte and to enhance their functional recovery in MDS, these factors may be powerful agents against infections when they are carefully used with regard to the activation of leukemic clones.

Adult↗