Comparison of long-term and short-term administration of itraconazole for primary antifungal prophylaxis in recipients of allogeneic hematopoietic stem cell transplantation: a multicenter, randomized, open-label trial

2014 ◽  
Vol 16 (2) ◽  
pp. 286-294 ◽  
Author(s):  
R. Lin ◽  
X. Xu ◽  
Y. Li ◽  
J. Sun ◽  
Z. Fan ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Antifungal Prophylaxis ◽  
Short Term ◽  
Open Label ◽  
Hematopoietic Stem ◽  
Term Administration ◽  
Open Label Trial

The Comparison Between Long-Term and Short-Term Administration of Itraconazole for Primary Antifungal Prophylaxis in Recipients of Allogeneic Hematopoietic Stem Cell: A Multicenter, Randomized, Open-Label Trial (NCT01160952)

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 1930-1930
Author(s):  
Ren Lin ◽  
Yonghua Li ◽  
Xiaojun Xu ◽  
Zhiping Fan ◽  
Qianli Jiang ◽  
...  
Keyword(s):  
Stem Cell ◽  
Fungal Infection ◽  
Antifungal Prophylaxis ◽  
Short Term ◽  
Open Label ◽  
Hematopoietic Stem ◽  
Term Administration ◽  
Intent To Treat ◽  
Wbc Count

Abstract Abstract 1930 Background: Invasive fungal infection (IFI) is a major cause of death in recipients of allogeneic hematopoietic stem cell transplant (allo-HSCT). Although antifungal prophylaxis has been demonstrated effective for prevention of IFI after allo-HSCT, the optimal agents and the term of prophylaxis remain a matter of discussion. To investigate the effect of prophylaxis term for IFI after allo-HSCT, this multicenter, randomized, open-label study was conducted to compare the efficacy and safety between long-term and short-term administration of itraconazole for primary antifungal prophylaxis in recipients of allo-HSCT. Methods: A total of 128 patients without a history of IFI were enrolled in this study. The primary antifungal prophylaxis was initiated when WBC count < 1.0*10⋀9/L or on day 0. Itraconazole was administered intravenously with a loading dose of 400 mg·d−1for 2 days followed by 200 mg·d−1and switched to itraconazole orally on day +14 or when WBC count > 1.0*10⋀9/L until 30 days post-HSCT in short-term arm or 90 days in long-term arm. The primary endpoint was the incidence of proven or probable IFI at 30 days and 90 days post-HSCT. Results: Of the 129 enrolled patients, date of 121 cases were used to determine the primary endpoint in the intent-to-treat population (59 for long-term and 62 for short-term) and 7 patients died of transplant complication or other causes before the use of itraconazole. The baseline demographic and transplant characteristic were similar in the two arms. The incidence of proven and probable IFI within 90 days post-transplants was 1.69%. Intent-to-treat analysis showed that there was not a significant difference in the rate of breakthrough IFI (proven or probable IFI) between he long-term and short-term arm (1.69% vs. 0%; P=0.311). The incidence of IFI (proven, probable, and possible) in long-term arm was higher than short-term arm within 90 days post-transplants (6.78% vs. 0%, P=0.03). Acute GVHD was the risk factor affected the occurrence of IFI after HSCT (OR= 2.750, 95%CI 1.939–3.901, P=0.023). Prophylaxis was interrupted in 11 patients due to event of intolerance, including 1 in the short-term arm and 10 in the long-term arm (P=0.004). The incidences of drug-related adverse events in the two arms were similar (6.78% vs. 3.23%, P=0.43). The incidence of overall survival at 6 months was 75.74% in long-term arm and 84.56% in short-term arm (P=0.459). Conclusions: This study indicates that itraconazole was effective for prevention of IFI within 30 days post-HSCT. Whether the prolonged term administration of antifungal agent for prevention fungal infection is benefit to the recipients in allo-HSCT is worthy of further exploration. This trial was registered at www.clinicaltrials.gov as NCT01160952. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Clinical and immunologic outcome of patients with cartilage hair hypoplasia after hematopoietic stem cell transplantation

Blood ◽  
2010 ◽  
Vol 116 (1) ◽  
pp. 27-35 ◽  
Author(s):  
Victoria Bordon ◽  
Andrew R. Gennery ◽  
Mary A. Slatter ◽  
Els Vandecruys ◽  
Genevieve Laureys ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Clinical Manifestations ◽  
Hematopoietic Stem ◽  
Cartilage Hair Hypoplasia ◽  
Severe Immunodeficiency

Abstract Cartilage-hair hypoplasia (CHH) is a rare autosomal recessive disease caused by mutations in the RMRP gene. Beside dwarfism, CHH has a wide spectrum of clinical manifestations including variable grades of combined immunodeficiency, autoimmune complications, and malignancies. Previous reports in single CHH patients with significant immunodeficiencies have demonstrated that allogeneic hematopoietic stem cell transplantation (HSCT) is an effective treatment for the severe immunodeficiency, while growth failure remains unaffected. Because long-term experience in larger cohorts of CHH patients after HSCT is currently unreported, we performed a European collaborative survey reporting on 16 patients with CHH and immunodeficiency who underwent HSCT. Immune dysregulation, lymphoid malignancy, and autoimmunity were important features in this cohort. Thirteen patients were transplanted in early childhood (∼ 2.5 years). The other 3 patients were transplanted at adolescent age. Of 16 patients, 10 (62.5%) were long-term survivors, with a median follow-up of 7 years. T-lymphocyte numbers and function have normalized, and autoimmunity has resolved in all survivors. HSCT should be considered in CHH patients with severe immunodeficiency/autoimmunity, before the development of severe infections, major organ damage, or malignancy might jeopardize the outcome of HSCT and the quality of life in these patients.

scholarly journals Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

Blood ◽  
2008 ◽  
Vol 111 (1) ◽  
pp. 439-445 ◽  
Author(s):  
Hulya Ozsahin ◽  
Marina Cavazzana-Calvo ◽  
Luigi D. Notarangelo ◽  
Ansgar Schulz ◽  
Adrian J. Thrasher ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Collaborative Study ◽  
Hematopoietic Stem ◽  
Long Term Outcome ◽  
Wiskott Aldrich Syndrome

Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency with microthrombocytopenia, eczema, recurrent infections, autoimmune disorders, and malignancies that are life-threatening in the majority of patients. In this long-term, retrospective, multicenter study, we analyzed events that occurred in 96 WAS patients who received transplants between 1979 and 2001 who survived at least 2 years following hematopoietic stem-cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections, and sequelae of before or after HSCT complications. Three patients (3%) died 2.1 to 21 years following HSCT. Overall 7-year event-free survival rate was 75%. It was lower in recipients of mismatched related donors, also in relation with an older age at HSCT and disease severity. The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status (P < .001), suggesting that residual-host lymphocytes can mediate autoimmune disease despite the coexistence of donor lymphocytes. Infectious complications (6%) related to splenectomy were also significant and may warrant a more restrictive approach to performing splenectomy in WAS patients. Overall, this study provides the basis for a prospective, standardized, and more in-depth detailed analysis of chimerism and events in long-term follow-up of WAS patients who receive transplants to design better-adapted therapeutic strategies.

scholarly journals Long-term follow-up of autologous hematopoietic stem cell transplantation for severe refractory Crohn's disease

2011 ◽  
Vol 5 (6) ◽  
pp. 543-549 ◽  
Author(s):  
Daniel W. Hommes ◽  
Marjolijn Duijvestein ◽  
Zuzana Zelinkova ◽  
Pieter C.F. Stokkers ◽  
Maartje Holsbergen-de Ley ◽  
...  
Keyword(s):  
Stem Cell ◽  
Crohn’S Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Long Term Follow Up
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