A pyruvate kinase deficiency child with novel PK‐LR gene mutations was successfully cured by matched unrelated donor peripheral blood stem cell transplantation

2021 ◽  
Author(s):  
Li‐ping Zhan ◽  
Li‐ping Que ◽  
Zheng‐zhou Wu ◽  
Dian‐dian Liu ◽  
Kai‐mei Wang ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Pyruvate Kinase ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Gene Mutations ◽  
Unrelated Donor ◽  
Matched Unrelated Donor ◽  
Pyruvate Kinase Deficiency ◽  
Blood Stem Cell Transplantation

Comparative Analysis of Outcomes of Allogeneic Peripheral Blood Stem Cell Transplantation From Related and Unrelated Donors for Adults with Hematologic Malignancies

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2302-2302
Author(s):  
Chunji Gao ◽  
Xiaohong Li ◽  
Honghua Li ◽  
Wenrong Huang ◽  
Xiaoxiong Wu ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Hematologic Malignancies ◽  
Blood Stem Cell ◽  
Unrelated Donor ◽  
Gvhd Prophylaxis ◽  
Blood Stem Cell Transplantation

Abstract Abstract 2302 Although allogeneic peripheral blood stem cell transplantation from a matched related donor (RD-PBSCT) presents the best curable opportunity for many patients with hematologic malignancies, only about one third of individuals have HLA matched sibling donors. Fortunately, from unrelated donor peripheral blood stem cell transplantation has been acceptable and expanded recently. In order to evaluate the effect of allogeneic peripheral blood stem cell transplantation from unrelated donor (URD-PBSCT), we compare results of URD-PBSCT and RD-PBSCT that were done for 172 consecutive adult patients with hematologic maligancies from Jan 2002 to Dec 2008 at a single-center. Among these patients, 62 cases underwent URD-PBSCT and 110 cases underwent RD-PBSCT. Myeloablative conditioning was adopted for all patients. In graft versus host disease (GVHD) prophylaxis, 49 URD-PBSCT recipients received CSA, MTX, MMF and ATG (URD-ATG group), 13 recipients were given simulect more in base of URD-ATG group (URD-ATG+CD25 group) while RD-PBSCT recipients (RD group) received CSA, MTX and MMF. Engraftment was achieved in 98.4% of URD-PBSCT patients and 98.2% of RD-PBSCT patients (100% for patients surviving beyond 28 days after transplant). The cumulative incidence of acute GVHD (grade II-IV) was 15.4%, 36.7% and 29.1% respectively in the URD-ATG+CD25, URD-ATG and RD groups (P = 0.275). The cumulative incidence of chronic GVHD was 0%, 45.6%, 39.6% respectively and significant lower in URD-ATG+CD25 group than the other two groups (P = 0.0134). Relapse incidence was 53.8%, 12.2%, 14.5% respectively and significant higher in URD-ATG+CD25 group than the other s (P = 0.0059), while there was no different between the URD-ATG and RD groups (P = 0.610). Estimated overall survival (OS) at 5 years was 30.8%, 69.4% and 67.3% respectively and no significant difference between URD-ATG group and RD group (p=0.898). Adverse prognosis factors for relapse and OS included transplant not in remission and GVHD prophylaxis with simulect. Our results indicate PBSCT from unrelated donor may be considered comparable to those from related donor. Disclosures: No relevant conflicts of interest to declare.

Low-Dose Azacitidine for Relapse of MDS/AML after Unrelated Donor Peripheral Blood Stem Cell Transplantation.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 5034-5034 ◽  
Author(s):  
James M. Rossetti ◽  
Richard K. Shadduck ◽  
Chandana Thatikonda ◽  
Entezam Sahovic ◽  
John Lister
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Low Dose ◽  
Peripheral Blood Stem Cell ◽  
Unrelated Donor ◽  
Post Transplant ◽  
Blood Stem Cell Transplantation

Abstract Background: Post-transplant relapse in patients with myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) is difficult to manage. Cytogenetic relapse and decreasing donor chimerism often precedes morphological relapse. Weaning of immunosuppressive agents and donor lymphocyte infusion (DLI) may induce graft versus tumor effect (GVT), but is of limited value for patients with existing GVHD. In addition, the degree of GVT in high-risk myeloid disease is suboptimal. We present our experience using low-dose azacitidine (AZA) for cytogenetic relapse post-transplantation. Methods: Six patients with high-risk myeloid malignancy with cytogenetic relapse after matched unrelated donor peripheral blood stem cell transplantation were treated with low-dose AZA at 25 mg/m2 SC or IV for 5 days. An average of 2 cycles of AZA were given (range = 1 to 3). There were 3 females and 3 males with a mean age of 48 years (range = 31 to 59 years). Four had high-grade MDS (including 2 with treatment related disease) and 2 had high-risk AML. Conditioning consisted of fludarabine and ablative doses of busulfan in all patients. Cytogenetic relapse was seen by FISH testing within 187 days post-transplant (range = 30 to 730 days). AZA was given after an initial attempt to wean immunosuppression, which was not possible in 3 patients due to existing GVHD. All patients tolerated AZA well, without major toxicity. DLI was possible in 3 patients following AZA. A reduction in cytogenetic abnormalities (by FISH) and increase in donor chimerism (by FISH or STR) was observed in 5 out of 6 patients (83%) within 21 days post-AZA (range = 7 to 71 days). Three of the 5 responders demonstrated improvement after 1 cycle. The other 2 responders improved after 2 cycles given 28 and 60 days apart, respectively. One of these patients responded to a second AZA cycle after failing DLI. AZA did not appear to induce or worsen GVHD in any patient. One patient remains in CR 4 months after 1 cycle of AZA. Another patient demonstrated ongoing improvement in chimerism until her death from previously existing GVHD 20 days after a third cycle of AZA. The remaining 3 responders relapsed within 30 days from time of first response (range = 17 to 43 days). Conclusions: Low-dose AZA appears to have activity in post-transplant relapse of MDS and AML. This low-dose regimen appears to be well tolerated, however, response to AZA is short-lived in the majority of patients. Further investigation is planned to improve the durability of response by giving AZA at regular intervals from the time of early relapse. The utility of AZA as a preparatory regimen pre-DLI should also be explored.

scholarly journals Unmanipulated Peripheral Blood Stem Cell Transplantation with non-TBI Myeloablative Conditioning Regimen from Haploidentical and Unrelated versus Related Donors for Acute Leukemia in Children, Adolescents and Young Adults (CAYA): A Competing Risk Analysis

2021 ◽  
Author(s):  
Seied Asadollah Mousavi ◽  
Tahereh Rostami ◽  
Azadeh Kiumarsi ◽  
Amir Kasaeian ◽  
Mohammadreza Rostami ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Acute Leukemia ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Conditioning Regimen ◽  
Unrelated Donor ◽  
Unrelated Donors ◽  
Blood Stem Cell Transplantation

Abstract BackgroundAllogeneic hematopoietic stem cell transplantation (HSCT) is the only potentially curative treatment for acute leukemia. Many different parameters have significant impact on the final results of HSCT such as donor type, stem cell source, and the implemented conditioning regimen. In the absence of an HLA-matched related donor, unrelated donors or haploidentical donors are possible alternatives for patients with an indication to HSCT. In order to compare the outcomes of HSCT from different donor types, in this single-center study, using a radiation-free MAC regimen, we compared the results of unmanipulated peripheral blood stem cell transplantation (PBSCT) from matched and mismatched related and unrelated donors with haploidentical donors in the children, adolescents and young adults (CAYA) affected by acute leukemia.MethodsIn this retrospective study, since 2014 to 2021, the outcome of CAYA patients with acute leukemia who had undergone peripheral blood T cell-replete HSCT from haploidentical donors versus unrelated donors (including 10/10 or 9/10 HLA-matched) versus related donors (including 10/10 or 9/10 HLA-matched) were evaluated. The HSCT was based on a radiation-free MAC regimen including Busulfan and Cyclophosphamide. The GvHD prophylaxis was based on the administration of Cyclosporine A in all patients, plus rabbit anti-human thymocytes globulins in unrelated and haploidentical donors and post transplantation cyclophosphamide in haploidentical donors. Adjusted multivariable proportional hazard Cox and competing risk analyses were performed.ResultsMedian follow up time was 28.7 months (95% CI: 21.9-34.9). Three-year overall survival rate (OS) and GvHD-free/relapse-free survival (GFRFS) rate was 68.81% (95% CI: 60.08%-76.01%) and 44.19% (95% CI: 35.52%-52.49%), respectively. Patients who had undergone HSCT from an unrelated donor had the lowest OS and GFRFS compared to other donor types. The 3-years NRM in all patients was 7.84% (95% CI 4.36-12.62). Adjusted multivariable modeling of OS showed that the hazard of death in patients who had undergone HSCT from an unrelated donor, was 3.6 times more than patients who underwent HSCT from their haploidentical donors (P=0.05). Likewise, the hazard of NRM after HSCT from unrelated donors was 6 times more than haploidentical donors (P=0.002). However, the relapse incidence was not significantly different between the two mentioned groups.ConclusionsIn this study, HSCT from haploidentical donors was associated with superior survival rates compared to HSCT from unrelated donors. So haploidentical peripheral blood derived HSCT could be a practical and valuable clinical option that offers CAYA patients with acute leukemia needing HSCT and lacking matched available donors, a reasonable opportunity for disease control.

A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation

Blood ◽  
2012 ◽  
Vol 120 (19) ◽  
pp. 3875-3881 ◽  
Author(s):  
Chunfu Li ◽  
Xuedong Wu ◽  
Xiaoqing Feng ◽  
Yuelin He ◽  
Huaying Liu ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Thalassemia Major ◽  
Blood Stem Cell ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Blood Stem Cell Transplantation

Abstract We used a novel NF-08-TM transplant protocol based on intravenous busulfan, cyclophosphamide, fludarabine, and thiotepa in 82 consecutive patients with β-thalassemia major (TM), including 52 with allogeneic peripheral blood stem cell transplantation (PBSCT) from unrelated donors (UDs) with well-matched human leukocyte antigens and 30 with hematopoietic stem cell transplantation (HSCT) from matched sibling donors (MSDs). The median age at transplantation was 6.0 years (range, 0.6-15.0 years), and the ratio of male-to-female patients was 56:26. The median follow-up time was 24 months (range, 12-39 months). The estimated 3-year overall survival and TM-free survival were 92.3% and 90.4% in the UD-PBSCT group and 90.0% and 83.3% in the MSD-HSCT group. The cumulative incidences of graft rejection and grades III-IV acute graft-versus-host disease were 1.9% and 9.6%, respectively, in the UD-PBSCT group and 6.9% and 3.6%, respectively, in the MSD-HSCT group. The cumulative incidence of transplant-related mortality was 7.7% in the UD-PBSCT group and 10.0% in the MSD-HSCT group. In conclusion, UD-PBSCTs using the well-tolerated NF-08-TM protocol show similar results to MSD-HSCTs and can be used to treat β-thalassemia patients in the absence of MSDs.

Sign in / Sign up

Export Citation Format

Share Document