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排序方式: 共有239条查询结果,搜索用时 15 毫秒
51.
Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome 总被引:8,自引:4,他引:8
Peters C; Balthazor M; Shapiro EG; King RJ; Kollman C; Hegland JD; Henslee- Downey J; Trigg ME; Cowan MJ; Sanders J; Bunin N; Weinstein H; Lenarsky C; Falk P; Harris R; Bowen T; Williams TE; Grayson GH; Warkentin P; Sender L; Cool VA; Crittenden M; Packman S; Kaplan P; Lockman LA 《Blood》1996,87(11):4894-4902
Long-term survival and improved neuropsychological function have occurred in selected children with Hurler syndrome (MPS I H) after successful engraftment with genotypically matched sibling bons marrow transplantation (BMT). However, because few children have HLA-identical siblings, the feasibility of unrelated donor (URD) BMT as a vehicle for adoptive enzyme therapy was evaluated in this retrospective study. Forty consecutive children (median, 1.7 years; range, 0.9 to 3.2 years) with MPS I H received high-dose chemotherapy with or without radiation followed by BMT between January 27, 1989 and May 13, 1994. Twenty-five of the 40 patients initially engrafted. An estimated 49% of patients are alive at 2 years, 63% alloengrafted and 37% autoengrafted. The probability of grade II to IV acute graft-versus-host disease (GVHD) was 30%, and the probability of extensive chronic GVHD was 18%. Eleven patients received a second URD BMT because of graft rejection or failure. Of the 20 survivors, 13 children have complete donor engraftment, two children have mixed chimeric grafts, and five children have autologous marrow recovery. The BM cell dose was correlated with both donor engraftment and survival. Thirteen of 27 evaluable patients were engrafted at 1 year following URD BMT. Neither T-lymphocyte depletion (TLD) of the bone marrow nor irradiation appeared to influence the likelihood of engraftment. Ten of 16 patients alive at 1 year who received a BM cell dose greater than or equal to 3.5 x 10(8) cells/kg engrafted, and 62% are estimated to be alive at 3 years. In contrast, only 3 of 11 patients receiving less than 3.5 x 10(8) cells/kg engrafted, and 24% are estimated to be alive at 3 years (P = .05). The mental developmental index (MDI) was assessed before BMT. Both baseline and post-BMT neuropsychological data were available for 11 engrafted survivors. Eight children with a baseline MDI greater than 70 have undergone URD BMT (median age, 1.5 years; range, 1.0 to 2.4 years). Of these, two children have had BMT too recently for developmental follow-up. Of the remaining six, none has shown any decline in age equivalent scores. Four children are acquiring skills at a pace equal to or slightly below their same age peers; two children have shown a plateau in learning or extreme slowing in their learning process. For children with a baseline MDI less than 70 (median age, 2.5 years; range, 0.9 to 2.9 years), post-BMT follow-up indicated that two children have shown deterioration in their developmental skills. The remaining three children are maintaining their skills and are adding to them at a highly variable rate. We conclude that MPS I H patients with a baseline MDI greater than 70 who are engrafted survivors following URD BMT can achieve a favorable long-term outcome and improved cognitive function. Future protocols must address the high risk of graft rejection or failure and the impact of GVHD in this patient population. 相似文献
52.
Acute thrombotic and hemorrhagic manifestations of thrombocytosis associated with myeloproliferative disorders may be life threatening. Conventional therapy with radioisotopes and/or cytotoxic drugs may require weeks for effective control of platelet counts. In five patients, plateletpheresis by discontinuous-flow (Haemonetics) or continuous-flow (Aminco Celltrifuge) centrifugation was used as a means of reducing platelet counts acutely. With each procedure, approximately 2-9 X 10(12) platelets were removed, resulting in decrements in platelet counts and relief of symptoms. Plateletpheresis is a useful and safe acute means of controlling platelet counts in myeloproliferative disorders. 相似文献
53.
Prostaglandin endoperoxide analogues stimulate phospholipase C and protein phosphorylation during platelet shape change 总被引:5,自引:0,他引:5
We have studied the effects of two stable prostaglandin endoperoxide analogues on platelet lipid metabolism in relation to specific platelet functional changes. During platelet shape change, the endoperoxide analogues induce the formation of 1,2-diacylglycerol and phosphatidic acid, indicating the activation of a phosphoinositide-specific phospholipase C. In parallel, they stimulate the phosphorylation of a 40-kd and a 20-kd protein. During platelet shape change, arachidonic acid is released, but not metabolized by platelet cyclo-oxygenase or lipoxygenase. Phospholipase C activation and platelet shape change are independent of extracellular Ca++ and Mg++, arachidonate metabolism, and release of adenosine diphosphate (ADP). Activation of phospholipase C during platelet aggregation seems, however, to be mediated partly by release of ADP. We conclude that endoperoxide analogues initially stimulate in platelets the formation of products derived from phospholipase C activation, which might serve as intracellular messengers for phosphorylation of specific proteins related to platelet shape change. 相似文献
54.
55.
EG Gkougkousis M Khan TR Terry JK Mellon 《Annals of the Royal College of Surgeons of England》2009,91(6):532-534
Three cases of recurrent post-coital haematuria are described. Extensive protracted investigations pinpointed urethral varicosities as the likely cause. All patients were successfully treated with diathermy fulguration. 相似文献
56.
Background
The use of restrictive measures such as quarantine draws into sharp relief the dynamic interplay between the individual rights of the citizen on the one hand and the collective rights of the community on the other. Concerns regarding infectious disease outbreaks (SARS, pandemic influenza) have intensified the need to understand public perceptions of quarantine and other social distancing measures. 相似文献57.
58.
BACKGROUND AND OBJECTIVES: Unplanned excisions of soft-tissue sarcomas of the extremities occur commonly. Our goal was to evaluate the presence of residual disease, the treatment outcomes as they relate to local and distant recurrence and 5-year survival, and the limb functional outcomes in patients with unplanned sarcoma excision who were treated with re-excision and adjuvant therapy. METHODS: Between 1993 and 1999, 42 patients presented to our institution after unplanned excision of soft-tissue sarcomas. Of those 42 patients, 38 without gross residual disease or metastatic lesions formed the basis of this review. All 38 patients underwent revision wide excision; most (31) also received adjuvant therapy (radiation and/or chemotherapy). Clinical data were obtained from analysis of patient records and radiographic studies. Univariate analysis was performed with logistical regression, and multivariate analysis was performed with Cox modeling. RESULTS: The overall 5-year survival rate was 91.3% and the disease-free 5-year survival rate was 82.2%. Univariate analysis showed that stage-III disease (American Joint Committee on Cancer classification of soft-tissue sarcomas), lesions below the fascia, a histologic high-grade, and the development of organ metastasis were statistically significant factors for mortality. Stage-III disease also was significant for mortality on multivariate analysis. Only stage-III disease was significant for the development of local recurrence. Eighty-four percent of the patients had good to excellent functional outcomes. CONCLUSIONS: Re-excision with adjuvant therapy proved to be a safe and effective method for treating the disease and preserving limb function. 相似文献
59.
60.
The question of payment to egg donors has recently focused the attention of
both the Human Fertilisation and Embryology Authority (HFEA) and licensed
clinics. An acute shortage of egg donors and the rising costs of assisted
conception treatment are matters of grave concern to many patients. To
understand the emotional and social effects of egg sharing and egg
donation, we conducted a survey of attitudes in a group of women who had
some knowledge or experience of egg donation. A total of 750 questionnaires
were sent out of which 217 were returned within the specified time limit.
From these, 107 respondents had experience of egg donation and 110 had made
enquiries about donation. The data from these questionnaires were collated
and tabulated by the National Opinion Polls (NOP) Research Group. An
analysis of the data produced the following key findings: (i) donating or
sharing eggs is a social issue, 94% discuss it with
partners/family/friends; (ii) altruistic motives are not the prerogative of
non-patient volunteers-egg share donors felt that helping the childless was
as important as having a chance of in-vitro fertilization (IVF) for
themselves; (iii) the treatment procedure causes the most anxiety for egg
donors. The recipients were most concerned about delays, donor
characteristics and how the eggs were allocated; (iv) most respondents
(65%) with prior experience of egg sharing would do it again - 63% of egg
share donors, 72% of egg share recipients; (v) cash rewards to egg donors
and outright advertising for donors were rejected by 64 and 62% of the
sample respectively; and (vi) counselling was highly valued and there were
no instances of 'shattered lives' after treatment. The findings do not
support the recently announced intentions of the HFEA to disallow payment
to gamete donors on the grounds of devalued consent. There is no precedent
in modern medicine for egg sharing. The patients surveyed drew a clear
distinction between egg sharing and financial rewards. As long as egg
donation is not covered by the National Health Service, it is fairer to
offer egg sharing than to refuse treatment to those unable to pay.
相似文献