Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0026986 (myelodysplastic syndrome)
 14,926 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Umbilical cord-blood (CB) has been used as a source of hematopoietic stem cells in pediatric patients with sibling donors. As a result of the success with CB transplantation from sibling donors, pilot programs for the banking of unrelated donor CB were initiated in the organization of Kanagawa Cord Blood Bank, Japan in 1995. As of December 1997, unrelated donor CB was used to reconstitute hematopoiesis in seven patients aged 0.7-12.8 years, weighing 7-36 kg with high-risk leukemia (n = 5), myelodysplastic syndrome (n = 1), and immunodeficiency syndrome (n = 1). Engraftment of CB was achieved in six patients. The absolute neutrophil count reached 500/microliter in a median of 27 days; a platelet count of 20,000/microliter was reached by a median of 64 days in three patients who could be evaluated. Five patients are currently surviving. Grade I GVHD developed in three patients and grade III in one patient; no GVHD developed in three patients. Although only a small number of patients have been studied and the period of observation is too short to determine long-term survival, HLA-matched or HLA-mismatched CB from unrelated donors can provide an alternative source of hematopoietic reconstitution for clinical transplantation.
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PMID:Unrelated umbilical cord-blood stem cell transplantation: a report from Kanagawa Cord Blood Bank, Japan. 980 77

We analyzed results of 40 infants less than 2 years of age who received bone marrow transplants (BMT) between May 1974 and January 1995 for treatment of acute myelogenous leukemia (AML; N = 34) or myelodysplastic syndrome (MDS; N = 6) to determine outcome and survival performance. Among the AML patients, 13 were in first remission, 9 were in untreated first relapse or second remission, and 12 were in refractory relapse. Patients were conditioned with cyclophosphamide in combination with either total body irradiation (TBI; N = 29) or busulfan (N = 11). Source of stem cells included 6 autologous donors, 15 HLA genotypically identical siblings, 14 haploidentical family members, and 5 unrelated donors. Graft-versus-host disease (GVHD) prophylaxis was methotrexate (MTX) for 17, MTX plus cyclosporine (CSP) for 14, or CSP plus prednisone for 3. Incidence of severe (grade 3-4) regimen-related toxicity was 10% and transplant-related mortality was 10%. Acute GVHD (grades II-III) occurred in 39% of allogeneic patients, and chronic GVHD developed in 40%. Relapse, the most significant problem for patients in this study, occurred in 1 MDS patient and 23 AML patients and was the cause of death for 19 patients. The 2-year probabilities of relapse are 46%, 67%, and 92%, respectively, for patients transplanted in first remission, untreated first relapse or second remission, and relapse. One MDS and 8 AML patients received second marrow transplants for treatment of relapse, and 5 of these survive disease-free for more than 1.5 years. All 6 MDS patients and 11 of 34 AML patients survive more than 1.5 years later. The 5-year probabilities of survival and disease-free survival are 54% and 38% for patients transplanted in first remission and 33% and 22% for untreated first relapse or second remission. None of the patients transplanted with refractory relapse survive disease-free. Outcome was significantly associated with phase of disease at transplantation and pretransplant diagnosis of extramedullary disease. Long-term sequelae included growth failure and hormonal deficiencies. Survival performance was a median of 100% (80% to 100%) and neurologic development for all survivors was appropriate for age. This study indicates that infants with AML have similar outcome after BMT compared with older children and that BMT should be performed in first remission whenever possible. In addition, allogeneic BMT provides effective therapy for the majority of infants with MDS.
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PMID:Bone marrow transplantation for children less than 2 years of age with acute myelogenous leukemia or myelodysplastic syndrome. 980 46

Thirty patients (median age of 32 years; range, 6-61) with hematologic disorders received unmanipulated peripheral blood stem cell transplants from HLA-matched or one-antigen-mismatched related donors following myeloablative therapy for acute lymphoblastic leukemia (7), acute myelogenous leukemia (6), chronic myelogenous leukemia (8), myelodysplastic syndrome (3), or other disorders (6). Granulocyte colony stimulating factor (G-CSF) mobilized peripheral blood stem cells were collected from donors in 1 to 3 aphereses. The apheresis products contained mean counts of 11.3 x 10(8) (range, 3.8-17.2) nucleated cells/kg and 6.7 x 10(6) (range, 1.3-16.7) CD34+ cells/kg. Graft-versus-host-disease (GVHD) prophylaxis consisted of cyclosporin A plus methotrexate, or FK506 plus methotrexate. All patients received G-CSF following their transplant. Although 1 patient died of pneumonia 6 days after transplantation, the others demonstrated rapid engraftment. Median days to recovery to 500/microliter neutrophils and 20,000/microliter platelets were 13 (range, 8-21) and 14 (range, 1-23) days, respectively. The incidence of acute GVHD grade II-IV was 33%; chronic GVHD developed in 57% of the assessable patients. There were no episodes of graft failure or rejection. Nineteen patients (63%) were alive and in complete remission from 147 to 839 days following their transplant (median follow-up of 560 days). Further follow-up study will be required to assess the incidence of chronic GVHD and graft-versus-leukemia (GVL) effects.
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PMID:[Allogeneic peripheral blood stem cell transplantation in 30 patients with hematologic disorders]. 986 19

Blood cell transplantation (BCT) is now common practice in the autologous setting. We performed a pilot study of allogeneic BCT, collected after the priming of an HLA-identical sibling with a glycosylated rhu-G-CSF (lenograstim) (10 microg/kg). Fifty-four patients were included (38 +/- 11; M/F = 33/21; CML (n = 17), AML (n = 14), ALL (n = 15); MDS (n = 8)). Transplant procedures were standard (TBI regimen = 47 (87%); MTX-CsA: n = 37; CsA-PDN: n = 17). No serious adverse events were reported in donors. A median of 11 (3.5-29.1) x 10(6)/kg CD34+ cells, 332 (33-820) x 10(6)/kg CD3+ cells were collected. Four patients did not engraft (early death: n = 2; graft failure: n = 2). Fifty-one patients initially recovered 0.5 x 10(9)/l ANC and 25 x 10(9)/l platelets at 15 (10-30) and 13 (9-188) days. 29/51 and 29/38 experienced grade > or =2 acute and chronic GVHD. With a median follow-up of 25 months (18-36), relapse rate is 16% +/- 8, survival and DFS probabilities are similar (50% +/- 13). A better outcome is documented for patients under 45 years and in the early phase of the disease (n = 28), with an identical survival and DFS of 71% +/- 13. In conclusion, lenograstim is a potent rhu-G-CSF for mobilisation of allogeneic hematopoietic progenitors. Two-year follow-up indicates good haematological recovery but some concerns about graft failure and chronic GVHD have arisen deserving prospective evaluation.
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PMID:Mobilisation of healthy donors with lenograstim and transplantation of HLA-genoidentical blood progenitors in 54 patients with hematological malignancies: a pilot study. 1045 58

Between February 1995 and August 1997, 11 children (eight males, three females) aged 4-16 years (median 7 years) underwent allogeneic PBPC transplantation for treatment of hematological disorders. Seven patients with acute leukemia (n = 5 ALL, n = 1 AML) or lymphoma (n = 1) received primary allogeneic PBPC transplantation, four patients received a second allotransplantation for graft failure (n = 1 AML, n = 1 sickle cell anemia) or disease recurrence (n = 1 ALL, n = 1 MDS). Five donors were HLA-identical siblings, five were 0-1 antigen mismatched family members and one was a matched unrelated donor. Donors received G-CSF 10-12 microg/kg/day for 3-7 days, and underwent one or two leukaphereses. The median cell yield per donor expressed per kg of recipient body weight was as follows: mononucleated cells 10.8 x 10(8)/kg (range 4.7-21.2); CD34+ cells 8.6 x 10(6)/kg (range 3.2-22); CD3+ cells 3.7 x 10(8)/kg (range 2.7-7.5). All patients achieved an ANC >0.5 x 10(9)/l after a median of 12 days (11-18). An unsupported platelet count >50 x 10(9)/l was reached 15 days (13-21) after PBPC transplantation; four patients failed to reach this threshold. Acute GVHD (aGVHD) grades II to IV occurred in eight (73%) patients: seven of them experienced grade III-IV aGVHD. Seven patients evaluable for chronic GVHD (cGVHD) were scored as absent in five, limited in one and extensive in one patient. As of September 1997, six patients (55 %) were alive between 60 and 938 days post-transplant (median follow-up 274 days); four patients with malignancy were alive in CR after primary allotransplantation, two patients were alive after a second PBPC transplant. Five patients have died with the main causes of death being aGVHD (n = 3), ARDS (n = 1), relapse of the underlying disease (n = 1). In conclusion, despite the limited number of patients, these preliminary results indicate that PBPC may be considered as an alternative to bone marrow for allografting also in children.
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PMID:Allogeneic transplantation of peripheral blood progenitor cells in children: experience of two pediatric centers. 998 87

Eleven children underwent BMT for therapy-related MDS or leukemia, four from HLA-identical siblings and seven from unrelated donors. Ten of the 11 were conditioned with busulfan and cyclophosphamide as the majority had received prior irradiation to the chest and/or abdomen. All patients engrafted. Regimen-related toxicity was more common when compared to historical controls. Eight patients developed acute GVHD and four of eight who survived 100 days post transplant developed extensive chronic GVHD. Non-relapse related mortality occurred in three patients. Five patients developed recurrent malignancy: one died from recurrence of osteosarcoma, three died of recurrent leukemia or MDS and another developed two subsequent malignancies (duodenal carcinoma and anaplastic astrocytoma). Three survive disease-free at 14+, 22+ and 43+ months for a 2 year actuarial cancer-free survival of 24% (95% confidence interval = 5-53%). Although allogeneic BMT can be curative, regimen-related toxicity is frequent and recurrent malignancy remains the major obstacle.
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PMID:Bone marrow transplantation in pediatric patients with therapy-related myelodysplasia and leukemia. 1003 46

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare clonal haematological disorder characterized by intravascular haemolysis and increased risk of thrombosis. PNH is associated with bone marrow failure syndromes including aplastic anaemia, myelodysplasia and leukaemia. Bone marrow transplants are sometimes used to treat PNH, but small series and reporting biases make assessment of transplant outcome difficult. The outcome of 57 consecutive allogeneic bone marrow transplants for PNH reported to the International Bone Marrow Transplant Registry (IBMTR) between 1978 and 1995 was analysed. The 2-year probability of survival in 48 recipients of HLA-identical sibling transplants was 56% (95% confidence interval 49-63%). Two recipients of identical twin transplants remain alive 8 and 12 years after treatment. One of seven recipients of alternative donor allogeneic transplants is alive 5 years after transplant. The most common causes of treatment failure were graft failure and infections. Our results indicate that bone marrow transplantation can restore normal bone marrow function in about 50% of PNH patients.
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PMID:Bone marrow transplants for paroxysmal nocturnal haemoglobinuria. 1005 Jul 24

Allogeneic peripheral blood stem cell transplantation (Allo-PBSCT) has in recent years become an alternative to allogeneic bone marrow transplantation because it facilitates rapid hematopoietic reconstitution without an increase in the incidence of severe graft-versus-host disease (GVHD). We report on a 61-year-old man with myelodysplastic syndrome (MDS) and myelofibrosis who received an allo-PBSCT from his HLA-matched 68-year-old brother. The preparative regimen consisted of busulfan and cyclophosphamide. Cyclosporin A and methotrexate were administered for GVHD prophylaxis. The donor was treated with granulocyte colony-stimulating factor (G-CSF) at a dose of 10 micrograms/kg/day subcutaneously for 4 consecutive days. A preparation of 4.04 x 10(6) CD34+ cells/kg recipient weight was collected in a single apheresis and infused immediately. Engraftment times to a neutrophil count greater than 500/microliter and platelet count greater than 2.0 x 10(4)/microliter were 15 days each. Acute GVHD of grade II developed, but was resolved with methylprednisolone. However, the patient died of thrombotic microangiopathy 97 days after his allo-PBSCT. Administration of G-CSF and apheresis in the donor were feasible and well tolerated. Allo-PBSCT may result in earlier engraftment and be especially beneficial to elderly patients with MDS.
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PMID:[Allogeneic peripheral blood stem cell transplantation in an elderly patient with myelodysplatic syndrome with myelofibrosis]. 1006 93

We treated 13 patients with morphologically advanced myelodysplastic syndrome using cytosine arabinoside and total body irradiation, followed by allogeneic marrow transplantation from HLA-identical sibling donors. Granulocyte colony-stimulating factor (G-CSF) was added to the preparative regimen to selectively increase chemosensitivity of leukaemic cells and to improve transplant outcome. No regimen-related deaths occurred, and no side-effects related to the addition of G-CSF were observed except for transient mild bone pain. At a median follow-up time of 39 months the projected 5-year disease-free survival and 5-year overall survival were 67.7% and 75.5%, respectively, with only one case showing cytogenetic relapse. The preparative regimen including G-CSF is feasible, and preliminary results seem to be encouraging. However, a larger trial is clearly warranted to evaluate its efficacy.
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PMID:Treatment of advanced myelodysplastic syndrome with a regimen including recombinant human granulocyte colony-stimulating factor preceding allogeneic bone marrow transplantation. 1008 96

The cornerstone of therapeutic management for most patients with myelodysplastic syndrome (MDS) is supportive care, mainly in view of the average advanced age in MDS and the poor response to therapy. Due to the lack of satisfactory therapies, allogeneic stem cell transplantation is today the treatment of choice in the majority of young patients with histocompatible siblings. Results of treatment with allogeneic stem cell transplantation varies considerably depending on the stage of disease at transplantation and various clinical factors, such as the presence of cytogenetic abnormalities, age, and the percentage of blasts in the bone marrow at transplantation. Most patients may benefit optimally from an allogeneic stem cell transplantation when the transplant is performed as soon as an HLA-identical family member has been identified. Progression to more advanced leukaemic conditions will be associated with a higher failure rate mainly due to an increased incidence of relapse after transplantation. Delay of the transplant may be justified in a minority of patients with refractory anaemia without cytopenias or complex cytogenetic abnormalities. Patients who lack an HLA-identical family donor may be transplanted with either autologous stem cells or alternative allogeneic donors. The results are less compared to those obtained with histocompatible sibling transplantation due to an increased risk of relapse after autologous stem cell transplantation or a higher treatment-related mortality after transplantation with genotypically non-identical donors.
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PMID:Stem cell transplantation in myelodysplastic syndromes. 1010 Dec 12


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