scholarly journals Haploidentical haematopoietic stem cell transplantation for malignant infantile osteopetrosis and intermediate osteopetrosis: a retrospective analysis of a single centre

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Guanghua Zhu ◽  
Ang Wei ◽  
Bin Wang ◽  
Jun Yang ◽  
Yan Yan ◽  
...  
Keyword(s):  
Stem Cell ◽  
Survival Rate ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Clinical Manifestations ◽  
Haematopoietic Stem Cell ◽  
High Survival

Abstract Objective To evaluate the clinical efficacy of haploidentical haematopoietic stem cell transplantation (haplo-HSCT) for the treatment of malignant infantile osteopetrosis (MIOP) and intermediate osteopetrosis. Methods Children with MIOP and IOP who underwent haplo-HSCT in Beijing Children’s Hospital, Capital Medical University, from January 2010 to May 2018 were retrospectively analysed. Data relating to the clinical manifestations, engraftment, and prognosis of the children were extracted from medical records. Results Twenty-seven patients, including 18 males and 9 females, with an onset age of 12 (0.04–72) months were enrolled in this study. The median time from diagnosis to transplantation was 4 (1–23) months. All patients received haplo-HSCT with a myeloablative conditioning regimen (including fludarabine, busulfan, and cyclophosphamide). Graft versus host disease (GVHD) prophylaxis was based on anti-human T lymphocyte porcine immunoglobulin/anti-human thymus globulin, methotrexate, and mycophenolate mofetil. The median observation time was 55.2 (0.3–126.2) months. By the end of follow-up, twenty patients survived and seven patients died. The 5 year overall survival rate was 73.9%. Stage I-II acute GVHD was observed in 20 patients, stage III GVHD in 1 patient and no patients had stage IV disease. Chronic GVHD was observed in 11 patients (40.7%) and was controlled by anti-GVHD therapy. Conclusions Haplo-HSCT was an effective treatment for MIOP and IOP, with a high survival rate and significantly improved clinical symptoms. For patients with a vision impairment before HSCT, the improvement was slow after transplantation. The incidence of GVHD was high but mild and was effectively controlled by appropriate treatment. These data indicated that haplo-HSCT was a feasible treatment for MIOP and IOP.

scholarly journals Haploidentical Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis and Intermediate Osteopetrosis:A Retrospective Analysis of a Single-Center

2021 ◽  
Author(s):  
Guanghua Zhu ◽  
Ang Wei ◽  
Bin Wang ◽  
Jun Yang ◽  
Yan Yan ◽  
...  
Keyword(s):  
Stem Cell ◽  
Survival Rate ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
High Survival ◽  
Hematopoietic Stem

Abstract ObjectiveTo evaluate the clinical efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for treatment of malignant infantile osteopetrosis (MIOP) and intermediate osteopetrosis.MethodsChildren with MIOP and IOP who underwent haplo-HSCT in Beijing Children’s Hospital, Capital Medical University, from January 2010 to May 2018 were retrospectively analyzed. Data relating to the clinical manifestations, engraftment, and prognosis of the children were extracted from medical records. ResultsTwenty-seven patients, including 18 males and 9 females, with an onset age of 12 (0.04-72) months, were enrolled in this study. The median time from diagnosis to transplantation was 4 (1-23) months. All patients received haplo-HSCT with myeloablative conditioning regimen (including fludarabine, busulfan, and cyclophosphamide). The graft versus host disease (GVHD) prophylaxis was based on anti-human T lymphocyte porcine immunoglobulin /anti-human thymus globulin, methotrexate, and mycophenolate mofetil. The median observation time was 55.2 (0.3-126.2) months. By the end of follow-up, twenty patients survived and seven patients died. The five years overall survival rate was 73.9%. Acute GVHD degree I-II was observed in 20 patients, degree III in 1 patient and without degree IV. Chronic GVHD was observed in 11 patients (40.7%). It was controlled by anti-GVHD therapy.ConclusionHaplo-HSCT was effective for MIOP and IOP, with high survival rate and significantly improved of clinical symptoms. For the patients with vision impairment before HSCT, the improvement was slow after transplantation. The incidence of GVHD was high but mild, and could be effectively controlled after appropriate treatment. These data provided that haplo-HSCT was feasible in the treatment of MIOP and IOP.

Engraftment after Haematopoietic Stem Cell Transplantation: Which Predictors Are Most Important?.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4947-4947
Author(s):  
Michael V. Lioznov ◽  
Raphael I. Ikogho ◽  
Boris Fehse ◽  
Nicolaus M. Kroeger ◽  
Axel R. Zander
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Conditioning Regimen ◽  
Haematopoietic Stem Cell ◽  
Platelet Recovery ◽  
Primary Disease ◽  
Multivariant Analysis

Abstract Rapid engraftment is one of the most important preconditions for successful haematopoietic stem cell transplantation (HSCT). Conditioning regimen, HSC dose, quality and source as well as primary disease and genetic disparities are possible parameters thought to be of crucial importance in this respect. In fact, more rapid engraftment after syngeneic vs. allogeneic transplantation of purified HSC was shown in the murine system (Uchida et al, 1998). However, an analogous influence of the HSCT type has not been systematically addressed in human HSCT yet. We retrospectively analysed leukocyte (>1,000/μl) and platelet (>20,000/μl) engraftment in relation to HSC and CFU dose, source as well as donor types and primary disease for 458 haematological patients who received HSC transplants after myeloablative full-conditioning. There was no statistically significant correlation between transplanted CD34+ cell number per kg patient’s body weight (BW) and day of both leukocyte (r=−0.04; p=0.18, Spearman rang-correlation) and platelet (r=−0.02; p=0.42) recovery. In contrast, we found a significant negative correlation (r=−0.26 for leukocyte and r=−0.21 for platelet; p<0.0001) between the numbers of transplanted CFU per kg BW and the day of engraftment. Transplanted CFU but not CD34+ numbers was also confirmed during multivariant analysis as independent prognostic factor for engraftment after HSCT. Engraftment occurred significantly (p<0.0001, Logrank-test) earlier after PBSCT (n=328) than after BMT (n=130) (day 13.7±3.3 vs. 16.7±4.2). Transplanted CFU numbers per kg BW were significantly (p<0.0001) different in the two groups: 26.3±21.0x105 per kg BW for CFU in the PBSCT group vs. 7.9±11.4x105 per kg BW correspondently in the BMT group. A causal independent role of transplanted CFU numbers per kg BW but not HSC source hold also true after multivariant analysis. Engraftment was seen significantly earlier after both syngeneic (n=9) as well as autologous (n=89) than after allogeneic (n=360) HSCT: at days 9.6±1.2 (syngeneic) and 11.5±1.7 (autologous) vs. day 15.9±4.0 (allogeneic; p<0.0001) for leukocytes and days 9.4±1.1 (syngeneic) and 12.6±2.2 (autologous) vs. 21.6±5.6 (allogeneic; p<0.0001) for platelets. The engraftment delay in autologous as compared to syngeneic HSCT is still significant (p<0.01) pointing to an influence of primary disease and/or previous chemotherapy cycles on the engraftment of autologous HSC. Importantly, there were no significant differences in transplanted CD34+ cell numbers and CFU per kg BW ih these three groups. Also, all above differences hold true when bone marrow and peripheral blood stem cell transplantations were analysed separately. Both uni- and multivariant analyses did not any dependence of leukocyte and platelet reconstitution kinetics on underlying diseases. The statistical uni- and multivariant analysis did not show any significant relations between donor or recipient age, donor or recipient gender, donor/recipient sex relations and speed of haematological recovery after HSCT. In conclusion our data clearly support a crucial independent role of the HSCT type and transplanted CFU numbers on the speed of both leukocyte and platelet recovery. Based on our results we suppose that genetic disparities seem to be the most important predictor of delayed engraftment and graft rejection and the use of functional assays (Aldefluor®, CFU) rather than phenotypical markers (CD34) is preferable for assessing HSC graft quality.

No Direct Effect of NOD2/CARD15 Variants On Clinical Outcome After Non-Myeloablative Allogeneic Stem Cell Transplantation.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4322-4322
Author(s):  
Hanneke M. van der Straaten ◽  
Martine M. Paquay ◽  
Marcel G.J. Tilanus ◽  
Leo F. Verdonck ◽  
Cynthia Huisman
Keyword(s):  
Stem Cell ◽  
Clinical Outcome ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Allogeneic Stem Cell Transplantation ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Haematopoietic Stem Cell ◽  
Significant Prognostic Factor ◽  
Wild Type

Abstract Abstract 4322 Background Single nucleotide polymorphisms (SNPs) in the innate immunity receptor NOD2/CARD15 have been demonstrated to modulate the outcome of allogeneic haematopoietic stem cell transplantation. The effect of the NOD2/CARD15 polymorphism seems to be associated with donor source as well as type of conditioning regimen. Methods We reviewed NOD2/CARD15 mutations in all donor/recipient pairs of 192 consecutive patients who received non-myeloablative allogeneic stem cell transplantation(SCT) at our institution between 2002 and 2006. All patients were treated uniformly with fludarabine 30 mg/m2/day for 3 days followed by 200 cGy TBI (n=154) or TBI alone (n=38) and received grafts from HLA-matched related (n=132) or unrelated (n=60) donors. Results Mutated alleles were observed in 36 of 192 (19%) patients and in 35 of 192 (18%) donors. These SNPs, however, did not have a significant impact on clinical outcome data (P > 0.05, Kaplan Meier and Fine & Gray's test). Acute graft-versus-host disease (GVHD) occurred in 24 of 61 (39%) patients with the polymorphism and in 66 of 131 (50%) patients without the polymorphism. Chronic GVHD developed in 28 of 55 (51%) patients with SNP pairs and in 79 of 121 (65%) patients with the wild type. The incidence of transplant-related mortality was 21% in both groups, 13 of 61 patients in the group with the polymorphism and 27 of 131 without the polymorphism. Relapse was seen in 23 of 61 (38%) patients with the SNP pairs and in 48 of 131 (37%) wild type patients. Finally, overall survival was 43% (26/61) in patients with the polymorphism and 39% (51/131) in patients without the polymorphism. Conclusion These data indicate that mutations in the NOD2/CARD15 genes do not influence the clinical outcome of non-myeloablative allogeneic SCT directly. Since NOD2/CARD15 variants are not recognized as a single significant prognostic factor, screening for NOD2/CARD15 when selecting a donor does not seem to have additional value in patients undergoing non-myeloablative SCT. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Refractory autoimmune haemolytic anaemia following allogenic haematopoietic stem cell transplantation: successful treatment of rituximab

2019 ◽  
Vol 47 (7) ◽  
pp. 3320-3331
Author(s):  
Xiaofan Li ◽  
Jiafu Huang ◽  
Zhijuan Zhu ◽  
Nainong Li
Keyword(s):  
Animal Model ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haemolytic Anaemia ◽  
Haematopoietic Stem Cell Transplantation ◽  
Chronic Gvhd ◽  
Autoimmune Haemolytic Anaemia ◽  
Haematopoietic Stem Cell ◽  
Anti Cd20

Objective To investigate the effectiveness and safety of rituximab in treating autoimmune haemolytic anaemia (AIHA) after allogeneic haematopoietic stem cell transplantation (allo-HSCT). Methods Patients with refractory AIHA following allo-HSCT were treated once-weekly with rituximab 375 mg/m2 for a total of four doses. In an animal study, recipient CB6F1 mice were conditioned with busulfan/fludarabine and transplanted with splenocytes and T-cell-depleted bone marrow from C57Bl/6 mice. In this animal model, anti-CD20 monoclonal antibody (mAb) was evaluated to see if it could prevent graft versus host disease (GVHD). GVHD was monitored by body weight loss, GVHD clinical scores and the survival of each group of mice. Histopathological analyses of the skin, intestine, liver and lung were used to analyse the severity of GVHD. Results After rituximab therapy, refractory AIHA was resolved in all four patients as shown by increased haemoglobin levels. B-cell proportions were reduced with a relative increase of the proportions of T-cells following rituximab treatment. None of the four patients experienced chronic GVHD. In the animal model, anti-CD20 mAb treatment reduced GVHD. Conclusions Rituximab therapy deserves consideration for the treatment of post-HSCT patients with refractory AIHA. Further studies are needed to define the therapeutic role of this anti-CD20 mAb.

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