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 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.

Comparable Survival and Transplant-Related Mortality Following Allogeneic Hematopoietic Cell Transplantation from HLA Allele-Matched Unrelated and HLA-Identical Sibling Donors.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3146-3146 ◽  
Author(s):  
Thai M. Cao ◽  
Schickwann Tsai ◽  
Linda Kelley ◽  
Stephen C. Alder ◽  
Thomas C. Fuller ◽  
...  
Keyword(s):  
Overall Survival ◽  
Cell Transplantation ◽  
Acute Gvhd ◽  
Hematopoietic Cell Transplantation ◽  
Chronic Gvhd ◽  
Hematopoietic Cell ◽  
Allogeneic Hematopoietic Cell Transplantation ◽  
P Value ◽  
Unrelated Donor ◽  
Related Mortality

Abstract Comprehensive analyses of unrelated donor (URD) and recipient HLA-matching for allogeneic hematopoietic cell transplantation (AHCT) have demonstrated better outcomes when allele typing is performed using high-resolution nucleotide sequence-based techniques. To evaluate survival following myeloablative AHCT using allele-level HLA-matched URD as compared with HLA-identical sibling donors, we analyzed outcomes for 430 patients treated at our center between March 1991 and April 2005. Sequence-based allele typing was retrospectively performed for HLA-A, B, C, DR and DQ when not done at time of AHCT for URD (n = 124; 29%) and non-sibling related donors (n = 19; 4%). Donors were HLA-identical siblings (n = 276; 64%), HLA allele-matched URD (n = 52; 12%), single HLA-locus mismatched donors (n = 52; 12%), or > 1 locus mismatched donors (n = 50; 12%). The median age at transplant was 23.4 years (range: 0.2 – 61). The most common diagnoses were AML (n = 107; 25%), CML (n = 90; 21%), ALL (n = 86; 20%) and MDS (n = 50; 12%). Total body irradiation-based preparative regimens were used for 283 patients (66%). Bone marrow (BM) was the graft for 388 patients (90%) and GCSF-mobilized peripheral blood stem cells (PBSC) for the remaining 42 (10%). Graft-versus-host disease (GVHD) prophylaxes were cyclosporine and methotrexate (n = 327; 76%), long methotrexate (n = 42; 10%), T-cell depletion (n = 19; 4%), or other regimens (n = 42; 10%). With a median follow-up of 4.8 years (range: 0.2 – 12.1), the 5-year estimate of overall survival (OS) for the entire group was 48.2% (95% CI: 45.7 – 50.7) and transplant-related mortality (TRM) was 31.4% (95% CI: 28.8 – 34). As shown in the Table, OS and TRM were indistinguishable between AHCT performed with HLA-identical siblings compared with HLA allele-matched URD. There was also no difference in grade III – IV acute GVHD (P = .46) between these two groups whereas there was a trend towards more extensive chronic GVHD (HR 1.8; 95% CI: 0.9 – 3.6; P = 0.12) for the URD recipients. Using a multivariate analysis to adjust for advanced disease, age (> vs ≤ 30 years), graft (BM vs PBSC) and female-to-male gender mismatch, there remained no difference in OS between HLA-identical siblings and HLA allele-matched URD (P = 0.67). These results demonstrate that key outcomes (OS, TRM, and severe acute GVHD) are equivalent in recipients of grafts from either allele-level 10/10 HLA-matched URD or HLA-identical siblings. Overall Survival TRM Number Hazard Ratio 95% CI P value Hazard Ratio95% CI P value HLA-ID Sibling 276 1 - - 1 - - HLA-ID URD 52 1.1 0.7 – 1.7 0.67 0.8 0.4 – 1.6 0.58 1 Locus MM 52 1.3 0.9 – 2.0 0.19 1.4 0.8 – 2.4 0.25 > 1 Locus MM 50 2.0 1.4 – 2.9 < 0.001 2.6 1.7 – 4.1 < 0.001

scholarly journals Comparable Leukemia-Free Survival after Autologous Hematopoietic Cell Transplantation Versus HLA-Identical Sibling Hematopoietic Cell Transplantation for Adult Acute Myeloid Leukemia in First Complete Remission: A Registry Study By the Adult AML Working Group of the Japan Society for Hematopoietic Cell Transplantation

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2514-2514
Author(s):  
Motonori Mizutani ◽  
Masahiko Hara ◽  
Hiroyuki Fujita ◽  
Jun Aoki ◽  
Heiwa Kanamori ◽  
...  
Keyword(s):  
Acute Myeloid Leukemia ◽  
Complete Remission ◽  
Cell Transplantation ◽  
Myeloid Leukemia ◽  
Hematopoietic Cell Transplantation ◽  
Conflicts Of Interest ◽  
Hematopoietic Cell ◽  
Free Survival ◽  
First Complete Remission ◽  
Acute Myeloid

Abstract Allogeneic hematopoietic cell transplantation (HCT) from a matched sibling donor (MSD) may provide a cure for acute myeloid leukemia in first complete remission (AML/CR1), although the procedure is associated with a higher rate of treatment-related mortality (TRM) than autologous HCT, and it remains uncertain which modality is preferable as post-remission treatment. We therefore conducted a retrospective registry-based analysis to compare the outcomes of patients with AML/CR1 receiving autologous peripheral blood (PB) grafts (n=398, median age: 47, range: 17-80) and those receiving allogeneic MSD bone marrow (BM) grafts (n=633, median age: 38, range: 16-73) or allogeneic MSD PB grafts (n=475, median age: 42, range: 16-74) between 1995 and 2011. Consequently, the 5-year overall survival (OS) rates for the autologous PB, allogeneic BM and allogeneic PB recipients were 62% (95% confidence interval [CI], 57-67%), 61% (95% CI, 57-65%; P=0.90) and 54% (95% CI, 49-59%; P=0.07), respectively (Fig. 1-A), and the 5-year leukemia-free survival (LFS) rates were 57% (95% CI, 52-62%), 58% (95% CI, 54-63%; P=0.49) and 51% (95% CI, 46-56%; P=0.12), respectively(Fig. 1-B). Meanwhile, the 5-year cumulative incidence of TRM was 8% (95% CI, 5-11%), 16% (95% CI, 13-19%; P=0.009) and 19% (95% CI, 15-23%; P=0.0001), respectively, while that of relapse was 35% (95% CI, 30-40%), 26% (95% CI, 22-29%; P=0.003) and 30% (95% CI, 26-35%; P=0.08), respectively. A multivariate analysis performed with autologous PB HCT as the reference showed a hazard ratio (HR) for OS of 0.93 (95% CI, 0.73-1.18; P=0.53) for allogeneic BM HCT and 1.08 (95% CI, 0.83-1.39; P=0.57) for allogeneic PB HCT and an HR for LFS of 0.86 (95% CI, 0.69-1.09; P=0.21) and 0.98 (95% CI, 0.77-1.24; P=0.85), respectively. Stratifying the patients according to cytogenetics (favorable, intermediate and poor) and age (<50 years and ≥ 50 years) did not influence the results. Accordingly, autologous PB HCT may be a viable alternative as post-remission therapy in patients with AML/CR1 in the absence of MSD. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

scholarly journals Reducing the Risk for Transplantation-Related Mortality After Allogeneic Hematopoietic Cell Transplantation: How Much Progress Has Been Made?

2011 ◽  
Vol 29 (7) ◽  
pp. 805-813 ◽  
Author(s):  
John T. Horan ◽  
Brent R. Logan ◽  
Manza-A. Agovi-Johnson ◽  
Hillard M. Lazarus ◽  
Andrea A. Bacigalupo ◽  
...  
Keyword(s):  
Overall Survival ◽  
Complete Remission ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Allogeneic Hematopoietic Cell Transplantation ◽  
Unrelated Donor ◽  
Major Barrier ◽  
Relative Risks ◽  
Related Mortality

PurposeTransplantation-related mortality (TRM) is a major barrier to the success of allogeneic hematopoietic cell transplantation (HCT).Patients and MethodsWe assessed changes in the incidence of TRM and overall survival from 1985 through 2004 in 5,972 patients younger than age 50 years who received myeloablative conditioning and HCT for acute myeloid leukemia (AML) in first complete remission (CR1) or second complete remission (CR2).ResultsAmong HLA-matched sibling donor transplantation recipients, the relative risks (RRs) for TRM were 0.5 and 0.3 for 2000 to 2004 compared with those for 1985 to 1989 in patients in CR1 and CR2, respectively (P < .001). The RRs for all causes of mortality in the latter period were 0.73 (P = .001) and 0.60 (P = .005) for the CR1 and CR2 groups, respectively. Among unrelated donor transplantation recipients, the RRs for TRM were 0.73 (P = .095) and 0.58 (P < .001) for 2000 to 2004 compared with those in 1990 to 1994 in the CR1 and CR2 groups, respectively. Reductions in mortality were observed in the CR2 group (RR, 0.74; P = .03) but not in the CR1 group.ConclusionOur results suggest that innovations in transplantation care since the 1980s and 1990s have reduced the risk of TRM in patients undergoing allogeneic HCT for AML and that this reduction has been accompanied by improvements in overall survival.

scholarly journals Adolescents and Young Adults with Acute Myeloid Leukemia Are Associated with Higher Treatment-Related Mortality and Inferior Overall Survival after Allogeneic Hematopoietic Cell Transplantation Compared with Children

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4702-4702
Author(s):  
Daisuke Tomizawa ◽  
Shiro Tanaka ◽  
Tadakazu Kondo ◽  
Yoshiko Hashii ◽  
Yasuyuki Arai ◽  
...  
Keyword(s):  
Overall Survival ◽  
Acute Myeloid Leukemia ◽  
Cell Transplantation ◽  
Myeloid Leukemia ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Gvhd Prophylaxis ◽  
Treatment Related Mortality ◽  
Related Mortality ◽  
Acute Myeloid

Abstract BACKGROUND : There have been few reports on outcome of adolescent and young adults (AYAs) with acute myeloid leukemia (AML) who received allogeneic hematopoietic cell transplantation (allo-HCT). We performed a retrospective analysis of nationwide registration data of the Japan Society of Hematopoietic Cell Transplantation collected between 1990 and 2013 to assess the allo-HCT outcomes of AYA patients with AML in Japan. PATIENTS & METHODS : 2973 patients with de novo AML (excluding acute promyelocytic leukemia, myeloid leukemia associated with Down syndrome, and secondary AML) whose age was 0-29 years old at their first allo-HCT either in first or second remission (CR), primary induction failure, or first relapse were identified. Outcomes including overall survival rate (OS), disease-free survival rate (DFS), cumulative incidence of relapse (CIR), and treatment-related mortality (TRM) were compared between children (0-14 years old at HCT, N=1123) and AYAs (15-29 years old, N=1850). RESULTS : AYA patients had male predominance, higher incidence of intermediate risk cytogenetics but lower incidence of high-risk cytogenetics [-7/7q-, -5/5q-, complex karyotype, t(6;9), or t(16;21)], and higher prevalence of French-American-British M0, M1, and M2 morphology, while M5 and M7 were lower. Compared to children, AYA patients were transplanted more frequently by HLA-matched-related or unrelated donors, while less frequently by HLA-mismatched related or unrelated cord blood donors. As for conditioning regimen, proportion of myeloablative conditioning (MAC) using total-body-irradiation (TBI) was higher in the AYAs, while that of non-TBI MAC and reduced-intensity-conditioning (RIC) were lower. Cyclosporine was more frequently used than tacrolimus as graft-versus-host-disease (GVHD) prophylaxis in the AYAs. Five-year OS and DFS rates were significantly poorer in the AYAs: 58% vs 54% (p<0.01) and 53% vs 48% (p=0.03), respectively. Five-year CIR did not differ between the two groups; 33% vs 34% (p=0.99). However, 5-year TRM was significantly higher in the AYAs; 13% vs 16% (p=0.02). Multivariate analysis for both OS and TRM showed significant negative impact of AYAs, but not for DFS or CIR. Subgroup analyses showed that impact of AYAs for OS was greater in male, patients with low-risk cytogenetic abnormalities [t(8;21) or inv(16)], transplanted in CR, transplanted from HLA-matched related donor, received non-TBI MAC conditioning, or received cyclosporine-based GVHD prophylaxis. Finally, we analyzed the impact of transplant center types on HCT outcomes among the 317 adolescent patients (15-18 years old) who were transplanted at 1CR or 2CR, because they could be transplanted either by pediatricians or by adult hematologists according to their referral pattern. 92 cases were transplanted in pediatric centers, 203 in adult centers, and 22 in combined pediatric and adult centers. Basic characteristics and method of HCT did not differ significantly among the three groups. Interestingly, there was no difference in OS, DFS, CIR, or TRM. CONCLUSIONS : AYAs with AML showed inferior post-transplant survival, which was mainly due to higher TRM in the AYAs. Disclosures No relevant conflicts of interest to declare.

scholarly journals Trends in allogeneic stem cell transplantation for multiple myeloma: a CIBMTR analysis

Blood ◽  
2011 ◽  
Vol 118 (7) ◽  
pp. 1979-1988 ◽  
Author(s):  
Shaji Kumar ◽  
Mei-Jie Zhang ◽  
Peigang Li ◽  
Angela Dispenzieri ◽  
Gustavo A. Milone ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Allogeneic Hematopoietic Cell Transplantation ◽  
Unrelated Donor ◽  
Recent Cohort ◽  
Over Time ◽  
Treatment Related Mortality ◽  
Related Mortality

Abstract Allogeneic hematopoietic cell transplantation in multiple myeloma is limited by prior reports of high treatment-related mortality. We analyzed outcomes after allogeneic hematopoietic cell transplantation for multiple myeloma in 1207 recipients in 3 cohorts based on the year of transplantation: 1989-1994 (n = 343), 1995-2000 (n = 376), and 2001-2005 (n = 488). The most recent cohort was significantly older (53% > 50 years) and had more recipients after prior autotransplantation. Use of unrelated donors, reduced-intensity conditioning and the blood cell grafts increased over time. Rates of acute graft-versus-host (GVHD) were similar, but chronic GVHD rates were highest in the most recent cohort. Overall survival (OS) at 1-year increased over time, reflecting a decrease in treatment-related mortality, but 5-year relapse rates increased from 39% (95% confidence interval [CI], 33%-44%) in 1989-1994 to 58% (95% CI, 51%-64%; P < .001) in the 2001-2005 cohort. Projected 5-year progression-free survival and OS are 14% (95% CI, 9%-20%) and 29% (95% CI, 23%-35%), respectively, in the latest cohort. Increasing age, longer interval from diagnosis to transplantation, and unrelated donor grafts adversely affected OS in multivariate analysis. Survival at 5 years for subjects with none, 1, 2, or 3 of these risk factors were 41% (range, 36%-47%), 32% (range, 27%-37%), 25% (range, 19%-31%), and 3% (range, 0%-11%), respectively (P < .0001).

scholarly journals Impact of prior imatinib mesylate on the outcome of hematopoietic cell transplantation for chronic myeloid leukemia

Blood ◽  
2008 ◽  
Vol 112 (8) ◽  
pp. 3500-3507 ◽  
Author(s):  
Stephanie J. Lee ◽  
Manisha Kukreja ◽  
Tao Wang ◽  
Sergio A. Giralt ◽  
Jeffrey Szer ◽  
...  
Keyword(s):  
Chronic Myeloid Leukemia ◽  
Imatinib Mesylate ◽  
Cell Transplantation ◽  
Myeloid Leukemia ◽  
Hematopoietic Cell Transplantation ◽  
Chronic Phase ◽  
Hematopoietic Cell ◽  
Free Survival ◽  
Treatment Related Mortality ◽  
Related Mortality

AbstractImatinib mesylate (IM, Gleevec) has largely supplanted allogeneic hematopoietic cell transplantation (HCT) as first line therapy for chronic myeloid leukemia (CML). Nevertheless, many people with CML eventually undergo HCT, raising the question of whether prior IM therapy impacts HCT success. Data from the Center for International Blood and Marrow Transplant Research on 409 subjects treated with IM before HCT (IM+) and 900 subjects who did not receive IM before HCT (IM−) were analyzed. Among patients in first chronic phase, IM therapy before HCT was associated with better survival but no statistically significant differences in treatment-related mortality, relapse, and leukemia-free survival. Better HLA-matched donors, use of bone marrow, and transplantation within one year of diagnosis were also associated with better survival. A matched-pairs analysis was performed and confirmed a higher survival rate among first chronic phase patients receiving IM. Among patients transplanted with advanced CML, use of IM before HCT was not associated with treatment-related mortality, relapse, leukemia-free survival, or survival. Acute graft-versus-host disease rates were similar between IM+ and IM− groups regardless of leukemia phase. These results should be reassuring to patients receiving IM before HCT.

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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