Allogeneic stem cell transplantation for children with acquired severe aplastic anaemia: a retrospective study by the Viva-Asia Blood and Marrow Transplantation Group

2013 ◽  
Vol 162 (3) ◽  
pp. 383-391 ◽  
Author(s):  
Jing Chen ◽  
Vincent Lee ◽  
Cheng Juan Luo ◽  
Alan Kwok Shing Chiang ◽  
Suradej Hongeng ◽  
...  
Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 1419-1419 ◽  
Author(s):  
Kai Hübel ◽  
Olaf Weingart ◽  
Frauke Naumann ◽  
Julia Bohlius ◽  
Keith Wheatley ◽  
...  

Abstract Abstract 1419 Poster Board I-442 For more than 20 years high dose chemotherapy followed by allogeneic stem cell transplantation (SCT) has been considered as a reasonable approach for the treatment of patients with AML. Moreover, during the last decade new scientific and technical developments results in major changes of clinical practice of transplantation. Enhanced donor availabilities and new strategies, e.g. dose-reduced conditioning, now make allogeneic stem cell transplantation available to patients who do not have a related donor or would not tolerate high-dose chemotherapy due to age or comorbidities. Usually, the decision to start the work-up process for allogeneic transplantation in AML patients is based on the availability of a donor, the assignment to the cytogenetic risk group, and the response to induction therapy, as well as patient factors. However, there would be greater confidence in defining who should, or should not, receive an allograft if the available recommendations given in guidelines are consistent and similar. In this analysis, a comprehensive systematic literature search for best available evidence from controlled clinical trials was performed in the bibliographic databases MEDLINE, EMBASE and Cochrane Central. In addition, the websites of major organizations in Europe and the US (European Group for Blood and Marrow Transplantation, EBMT; European Society for Medical Oncology, ESMO; British Committee for Standards in Hematology, BCSH; American Society for Blood and Marrow Transplantation, ASBMT; National Comprehensive Cancer Network, NCCN) were screened and the specific databases of the National Guideline Clearinghouse and the Guideline International Network Database were also searched to identify the latest recommendations and guidelines. The following points were selected for systematic comparison of the best available evidence: Factors for risk assessment and categorization of AML, donor categories for allogeneic SCT (sibling donors / matched unrelated donors), allogeneic transplantation in first CR, allogeneic transplantation in relapse/progressive disease or second CR, and allogeneic transplants with reduced intensity conditioning regimen. Several interesting findings emerge from this analysis: 1) For patients with relapse or refractory disease donor availability should be explored and discussed, though this is not based on reliable evidence from randomized studies; 2) Patients in CR1 with intermediate or high risk disease who have a matched related donor available should receive allogeneic stem cell transplantation (intermediate risk; ASBMT: reasonable, NCCN: option); 3) For patients who lack a family donor the recommendations are not consistent; 4) Allogeneic transplantation with reduced conditioning in AML patients is feasible, but the superiority over standard therapeutic regimens has not been proven yet. In summary, current guidelines differ in critical points in the recommendation for allogeneic stem cell transplantation. Furthermore, it is likely that only well-defined subgroups of AML patients will benefit from stem cell transplantation. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 5046-5046
Author(s):  
Parvez Ahmed ◽  
Khalil Ullah ◽  
Tariq M. Satti ◽  
Shahid Raza ◽  
Qamar-Un-Nisa Chaudhry ◽  
...  

Abstract Allogeneic stem cell transplantation (SCT) from HLA matched sibling donor is the standard treatment option in younger patients with severe aplastic anaemia (SAA). In the current study outcome of 70 patients with SAA undergoing allogeneic SCT at our institution from July 2001 to June 2007 is presented. Median follow up time was 727 days (range 100–2187). Three patients received 2nd SCT due to graft failure or rejection so actual number of SCT in the patients was 73. Median age of the patients was 16 years (range 5–38), 55 males, and 15 females. Seventeen had major ABO mismatch while sex mismatch in the form of female donor to male patient was present in 23 cases, 7 had both major ABO and sex mismatch. Sixty four patients were CMV positive while 6 had CMV negative status. Conditioning regimens included; cyclophosphamide (Cy) 200 mg/kg with either antilymphocyte globulin (ALG) 45 mg/kg (n=33) or antithymocyte globulin (rabbit ATG) 11.25 mg/kg (n=26); Cy plus Campath 100 mg (n=6), fludarabine 150 mg/m2 plus Cy 300 mg/m2 and ATG (n=8). All patient undergoing 2nd transplant received conditioning with Cy, fludarabine and ATG. GVHD prophylaxis was given with cyclosporin (CSA) plus prednisolone (41) with or without short course of methotrexate (29). Patients received PBSC (10) or bone marrow (12) alone or both (48). Mean mononuclear and CD34+ cell doses were 5.59 x 108/Kg and 4.8 x 106/Kg respectively. Median time to neutrophil recovery was 11 days (range 7–24). Neutropenic fever was seen in 60% cases, with mean duration of fever being 8 days. In majority (66%) no focus of infection could be found. Various isolates included gram negative rods (n=6), staphylococcus (n=2) and fungi (n=5). Other post-transplant infections were tuberculosis (n=2), herpes zoster (n=2) and transfusion associated falciparum malaria (n=2). Post-BMT non-infectious complications included acute GVHD (24%), chronic GVHD (08%), hemorrhagic cystitis (14%), seizures (5%), and VOD (3%). Graft rejection and primary graft failure was seen in 3 and 2 cases respectively. Three of them received 2nd transplant and had successful recovery while the other 2 died of septicemia. Six patients died during peri-transplant period, 3 at day 100, and 8 beyond day 100. One patient died of unrelated cause at 2 years post-transplant. Main causes of death were septicemia (n=4), conditioning regimen toxicity (n=3), VOD (n=2), GVHD (n=2) and disseminated aspergillosis (n=2). The overall and disease free survival was 76%. In univariate analysis using Logrank and Wilcoxon test factors correlated with better survival were patient’s age <15 years, disease duration <6 months, previous transfusions <20 events, conditioning with fludarabine/Cy/ATG, and absence of chronic GVHD. SCT from HLA matched sibling donor is effective treatment modality in majority of the young patients with SAA.


Sign in / Sign up

Export Citation Format

Share Document