The potential role of unrelated donor cord blood transplantation (UD-CBT) in adults remains unclear. This study reports the results of UD-CBT in 22 adults with hematologic malignancies following conditioning with thiotepa, busulfan, cyclophosphamide, and antithymocyte globulin in 21, with thiotepa, fludarabine, and antithymocyte globulin in 1, and graft-versus-host disease (GVHD) prophylaxis with cyclosporine and prednisone. Median age was 29 years (range, 18-46 years), and median weight was 69.5 kg (range, 41-85 kg). HLA match was 6 of 6 in 1 case, 5 of 6 in 13 cases, and 4 of 6 in 8 cases.Median number of nucleated cells infused was 1.71 ؋ 10 7 /kg (range, 1.01 ؋ 10 7 /kg to 4.96 ؋ 10 7 /kg). All 20 patients surviving more than 30 days had myeloid engraftment, and only 1, who received the lowest cell dose, developed secondary graft failure. Median time to reach an absolute neutrophil count of at least 0.5 ؋ 10 9 /L was 22 days (range, 13-52 days). Median time to platelets numbered at least 20 ؋ 10 9 /L was 69 days (range, 49-153 days). Seven patients (32%) developed acute GVHD above grade II, and 9 of 10 patients at risk developed chronic GVHD, which became extensive in 4 patients.Twelve patients remained alive and diseasefree 3 to 45 months after transplantation. Disease-free survival (DFS) at 1 year was 53%. Age strongly influenced DFS (P ؍ .01). For patients aged 30 years or younger, the DFS at 1 year was 73%. These preliminary results suggest that UD-CBT should be considered a reasonable alternative in young adults with hematologic malignancy and no appropriate bone marrow donor. (Blood. 2001;98:2332-2338)
The benefit of azacitidine treatment in survival of high-risk myelodysplastic syndromes (MDS) patients compared with conventional care treatment (CCT) has not been established outside clinical trials. To assess its effectiveness, we compared overall survival (OS) between azacitidine and conventional treatment (CCT) in high-risk MDS patients, excluding those undergoing stem cell transplantation, submitted to the Spanish MDS registry from 2000 to 2013. Several Cox regression and competing risk models, considering azacitidine as a time-dependent covariate, were used to assess survival and acute myeloblastic leukemia (AML) progression. Among 821 patients included, 251 received azacitidine. Median survival was 13.4 (11.8-16) months for azacitidine-treated patients and 12.2 (11-14.1) for patients under CCT (P=0.41). In a multivariate model, age, International prognostic scoring system and lactate dehydrogenase were predictors of OS whereas azacitidine was not (adjusted odds ratio 1.08, 95% confidence interval 0.86-1.35, P=0.49). However, in patients with chromosome 7 abnormalities, a trend toward a better survival was observed in azacitidine-treated patients (median survival 13.3 (11-18) months) compared with CCT (median survival 8.6 (5-10.4) months, P=0.08). In conclusion, our data show that, in spite of a widespread use of azacitidine, there is a lack of improvement in survival over the years. Identification of predicting factors of response and survival is mandatory.
Donors' age, with a threshold of 38 years or more, and the rHuG-CSF schedule are the factors that significantly affected CD34+ cell mobilization and collection in healthy donors.
The administration of rHuG-CSF to donors <18 years old leads to CD34+ cell mobilization in a pattern similar to that observed in adults. Greater age was associated with a more frequent requirement for more than one apheresis to achieve a similar number of CD34+ cells.
Summary:A Spanish National PBPC Donor Registry has recently been established for short-and long-term safety data collection in normal donors receiving rhG-CSF. To date, 466 donors have been included in the Registry. Median (range) dose and duration of rhG-CSF administration was 10 g/kg/day (4-20) and 5 days (4-8), respectively. Donors underwent a median of two aphereses (range, 1-5). Adverse effects consisted mainly of bone pain (90.2%), headache (16.9%) and fever (6.1%), but no donor discontinued rhG-CSF prematurely due to toxicity. Side-effects were more frequent in donors receiving Ͼ10 g/kg/day than in those with lower doses (82.8% vs 61.8%; P = 0.004). A significant decrease between baseline and post-apheresis platelet counts was the most important analytical finding (229 ؋ 10 9 /l vs 140 ؋ 10 9 /l; P Ͻ 0.0001), with a progressive reduction in platelet count with each apheresis procedure. One donor developed pneumothorax that required hospitalization due to central venous line placement. The mean CD34 ؉ cell dose collected was 6.9 ؋ 10 6 /kg (range, 1.3-36), with only 14 donors (2.9%) not achieving a minimum target of CD34 ؉ cells of 2 ؋ 10 6 /kg. No definitive information about potential long-term side effects is yet available. However, we hope this National Registry will serve as a useful basis for better monitoring of the efficiency and side-effects of cytokine administration in healthy people. Keywords: granulocyte colony-stimulating factor; normal donors; stem cell mobilization; side-effects Recombinant human granulocyte colony-stimulating factor (rhG-CSF) is being widely used to treat neutropenia secondary to chemotherapy and to collect peripheral blood progenitor cells (PBPC) for use in autologous transplantation. [1][2][3] More recently, rhG-CSF has been given to healthy people for granulocyte collection, 4,5 and to mobilize and collect PBPC for allogeneic transplants. [6][7][8][9][10] In the allogeneic setting, the routine use of mobilized PBPC must be based not only on clinical results, but also on the safety of donors. remain important issues. Regarding this, the optimal dose and schedule of rhG-CSF for mobilization of PBPC have not been definitively established, and specific information about short-and long-term biological and clinical effects of rhG-CSF in normal donors is still limited. Thus, a prolonged follow-up of donors is clearly needed in order to rule out the development of toxicity and to ensure that administration of this cytokine to healthy individuals is completely devoid of long-term side-effects. This task will be more easily approached by the development of PBPC national or international donor registries that allow the inclusion of higher number of donors. 11,12 In Spain, a National PBPC Donor Registry has recently been developed to address potential adverse effects of the administration of rhG-CSF to normal donors for PBPC mobilization and collection. The objectives of this registry include a standardized data collection for the different centers administering rhG-CSF to normal d...
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