scholarly journals A Novel Protocol Decreased Incidence of Hepatic Veno-Occlusive Disease and Improved Results in Hematopoietic Cell Transplantation for Patients with b-Thalassemia Major

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5858-5858
Author(s):  
Chunfu Li ◽  
Fuyu Pei ◽  
Qi Li ◽  
Jianyun Liao ◽  
Shengli An ◽  
...  
Keyword(s):  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Conflicts Of Interest ◽  
Thalassemia Major ◽  
Occlusive Disease ◽  
Hematopoietic Cell ◽  
Common Complication ◽  
Alternative Donor ◽  
Matched Sibling ◽  
D Dimer

Abstract Hepatic veno-occlusive disease (VOD) is a common complication of hematopoietic cell transplantation (HCT), especially patients with β-thalassemia major (TM). To estimate whether incidence of VOD recently decreased after using NF-08-TM protocol (NP), 311 TM-HCTs performed from February 2003 to June 2013 were analyzed. 241 patients received NP in or after 2009 and 70 received non-NP before 2009. VOD was diagnosed by Seattle criteria (SC) or Baltimore Criteria (BC). Patients were stratified by Nanfang (NF) criteria. A total of 31(10.0%) and 14 (4.5%) HCTs developed VOD (6 and 5 developed severe VOD) in SC and BC cohorts, respectively. The incidence of VOD was significantly lower in NP versus non-NP groups and in Class 2 versus Class 3 groups. Overall survival was significantly higher in NP versus non-NP cohorts. Rate of VOD in alternative donor transplant (ADT) was similar to that in matched sibling transplant (MST). Requirement of platelet and value of D-dimer significantly increased in the VOD patients. Our study showed the incidence of VOD significantly decreased in our center after using NP. ADT was similar to MST on rate of VOD. NF criteria of stratification can indicate occurrence of VOD. The SC can be more suitable criteria for early diagnosis. Disclosures No relevant conflicts of interest to declare.

scholarly journals Recently Improved Results of Hematopoietic Cell Transplantation in Thalassemia Patients in Asia

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1257-1257
Author(s):  
Chunfu Li ◽  
Vincent Lee ◽  
Shau Yin Ha ◽  
Hai Peng Lin ◽  
Anselm Lee ◽  
...  
Keyword(s):  
Overall Survival ◽  
Cell Transplantation ◽  
Graft Rejection ◽  
Hematopoietic Cell Transplantation ◽  
Conflicts Of Interest ◽  
Thalassemia Major ◽  
Hematopoietic Cell ◽  
Free Survival ◽  
Recent Cohort ◽  
Related Mortality

Abstract Objective Hematopoietic cell transplantation (HSCT) is not widely used for patients with b-thalassemia major (TM) so far because of transplant toxicities. Furthermore the tangible outcomes of contemporary HSCTs in Asia are unknown. Methods A newly founded consortium named the Viva-Asia BMT Group collected HSCT data retrospectively and investigated whether TM-HSCT outcomes had improved in recent years in Asia and whether an upper age limit can be determined for safe transplantation. Results From 1991 to 2012, 422 TM-HSCTs were performed among the 8 Asian centers; half (n=209) were before January 2009 (early cohort) and the other half (n=213) were performed thereafter (recent cohort). All major outcomes including overall survival (OS), TM-free survival (TFS), incidences of graft rejection (GR) and transplant-related mortality (TRM) improved significantly in the recent cohort (OS 93.4%, TFS 88.6%, GR 4.4% and TRM 6.6%) when compared with the earlier cohort (OS 84%, TFS 68.1%, GR 17.1% and TRM 16%). In the recent cohort, favorable OS, TFS, GR and TRM were observed with unrelated donors (n=168, 92.8%, 87.8%, 4.2% and 7.2%, respectively), with one-antigen mismatched parental donors (n=26,100%, 100%, 0% and 0%, respectively), and in patients younger than 10 years (n=173, 96%, 90.8%, 4.7% and 4.0%, respectively). TFS with unrelated cord blood (35.3%) was poor. Conclusion The excellent outcomes of contemporary TM-HSCT in general suggest that transplantation is a viable option for many Asian patients, particularly those younger than 10 years from areas with limited resources for chronic transfusion and iron chelation. Figure 1A OS, TFS, GR and TRM in (A) the total 422 patients, (B) the recent cohort, and (C) the earlier cohort. Abbreviations: GR, graft rejection; OS, overall survival; TFS, thalassemia major-free survival; TRM, transplant-related mortality. Figure 1A. OS, TFS, GR and TRM in (A) the total 422 patients, (B) the recent cohort, and (C) the earlier cohort. Abbreviations: GR, graft rejection; OS, overall survival; TFS, thalassemia major-free survival; TRM, transplant-related mortality. Figure 1B Figure 1B. Figure 1C Figure 1C. Disclosures No relevant conflicts of interest to declare.

scholarly journals Stem Cell Transplantation for Thalassemia Major from Alternative Donors: A Single Center Experience from India

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2298-2298 ◽  
Author(s):  
Chepsy C Philip ◽  
Amrith Mathew ◽  
Naveen Kakkar ◽  
Praveen Sobti ◽  
M Joseph John
Keyword(s):  
Stem Cell ◽  
Fungal Infection ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Conflicts Of Interest ◽  
Thalassemia Major ◽  
Unrelated Donor ◽  
Cell Transplant ◽  
Class Iii ◽  
Matched Sibling

Abstract Allogeneic stem cell transplant (aSCT) which remains the only curative option for patients with β thalassemia major (TM) is often limited by a lack of ideal donors. The role of alternative (related phenotypic donor- RD and Unrelated donor -UD) donor hematopoietic cell transplantation in thalassemia is not well established. Use of conventional myeloablation with busulphan and cyclophosphamide in alternative RDs (either phenotypically identical or 1-antigen-mismatched donors) has been associated with higher graft failure and acute graft versus host disease (aGvHD) prompting novel approaches (Gaziev et al., 2000, 2013). We undertook a retrospective analysis of patients who underwent aSCT from alternative donors in TM. From February, 2009 to June, 2016, 51 HLA matched transplants for TM was done at our center. Among them 19 (37.3%) used alternate donors. In this cohort, the median age was 9 years (range: 2-18) and there were 13 (68.4%) males. Thirteen (68.4%) were Pesaro Class III and 8 (42.1%) were in the Class III higher risk group as defined earlier (Mathews et al., 2007). All 19 received a treosulfan based regimen. PBSC (Peripheral Blood Stem Cell) was the source of stem cells in 78% and bone marrow in the rest. All patients received a cyclosporin (CSA) plus short course methotrexate GvHD prophylaxis regimen. There were 4 (12%) deaths. The major cause of death was fungal infection (3 had invasive fungal infection of whom 1 developed candidemia following a very late UD deferral). Of the alternative grafts 7 (36.8%) received RD grafts and 13 (68.4%) from UD. We compared the results of RD with HLA-matched sibling (matched sibling donors [MSDs]) aSCT in 32 patients and UD in 12 patients (Table 1). One (14.3%) patient developed aGvHD (Grade 2-4). The entire RD group had sustained engraftment. Rejection incidence was 0% in the RD, 8.3% in UD and 3.4% in MSD groups, with respective thalassemia-free survival probabilities at one year of 68.6% ±18.6%, 83.3% ±15.2%, 90.0% ±5.5% (P = .549) Fig 1. In conclusion, the present data shows that a treosulfan based reduced toxicity myeloablative regimen with a PBSC graft has the potential to allow patients to safely undergo aSCT from phenotypically identical RD. Disclosures No relevant conflicts of interest to declare.

scholarly journals HLA-haploidentical vs matched-sibling hematopoietic cell transplantation: a systematic review and meta-analysis

2019 ◽  
Vol 3 (17) ◽  
pp. 2581-2585 ◽  
Author(s):  
Mohamad A. Meybodi ◽  
Wenhao Cao ◽  
Leo Luznik ◽  
Asad Bashey ◽  
Xu Zhang ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Meta Analysis ◽  
Hematopoietic Cell ◽  
Free Survival ◽  
Relative Contribution ◽  
Haploidentical Hematopoietic Cell Transplantation ◽  
Matched Sibling ◽  
Outcome Differences

Abstract HLA haploidentical hematopoietic cell transplantation (haplo-HCT) using posttransplantation cyclophosphamide (PT-Cy) is an alternative strategy when a matched sibling donor (MSD) is not available. We performed a systematic review and meta-analysis to compare the outcomes of MSD vs haplo-HCT. Eleven studies (1410 haplo-HCT and 6396 MSD recipients) were meta-analyzed. All studies were retrospective and high quality, and 9 were multicenter. Haplo-HCT was associated with ~50% lower risk of chronic graft-versus-host disease (GVHD) (hazard ratio [HR], 0.55; 95% confidence interval [CI], 0.41-0.74), but higher risk of nonrelapse mortality (HR, 1.36; 95% CI, 1.12-1.66). Relapse, survival, acute GVHD, and GVHD-free relapse-free survival were not significantly different between the groups. Deciphering the relative contribution of PT-Cy and HLA disparity to the observed outcome differences between the groups requires further research.

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