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Severe aplastic anemia (SAA) is a severe bone marrow failure syndrome caused by multiple causes, which is clinically manifested with severe anemia, infection and bleeding. The complex pathogenesis of SAA has not been fully understood. SAA is characterized with acute onset, severe disease condition and rapid progression. At present, with the in-depth study of SAA and the improvement of diagnosis and treatment, the therapeutic strategy for SAA has been evolved from classical immunosuppressive therapy based on antithymocyte globulin and cyclosporine to the application of thrombopoietin receptor agonist and combined treatment based on allogeneic hematopoietic stem cell transplantation, which may promote the reconstruction of hematopoietic function of SAA patients to varying degree and significantly improve survival and clinical prognosis, becoming the research hotspot of SAA treatment. In this article, new advances in the treatment of SAA at home and abroad were reviewed.
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Objective:To explore the differences of immune reconstitution between peripheral blood stem cell transplantation and umbilical cord blood transplantation.Methods:A total of 300 patients (aged 18 (8, 33), 163 males and 137 females) with malignant hematological diseases who received allogeneic hematopoietic stem cell transplantation in the First Affiliated Hospital of University of Science and Technology of China from January 2018 to March 2020 were enrolled in this study, including 255 cases of umbilical cord blood transplantation and 45 cases of peripheral blood stem cell transplantation. Multicolor flow cytometry was applied to analyze lymphocyte subsets of the percentages and absolute counts in the two donor types and peripheral blood of patients after receiving hematopoietic stem cell transplantation. The differences between the two grafts were compared, and the lymphocyte subsets results were evaluated at 1, 2, 3, 4, 6, 9, 12, and 18 months after transplantation. 18-month disease-free survival (DFS) within the 300 patients under the two transplantation types were retrospectively analyzed.Results:1. The proportion of NKT cells in peripheral blood group was significantly higher than that in cord blood group (2.79% vs 0.24%, P<0.001). 2. The proportion of helper T cells in the UCBT group was higher than that in the PBSCT group, as well as the counts 6 months after transplantation ( P<0.05). 3. The proportion of NK1 cells (3 rd to 9 th month) and count (4 th to 12 th month) in UCBT group were significantly higher than those in PBSCT group ( P<0.05). 4. NKT cells in the UCBT group were lower than those in the PBSCT group (proportion and count) throughout the monitoring process ( P≤0.001). 5. The proportion of DNT cells (within 1 year) and count (within 6 months) in the UCBT group were significantly lower than those in the PBSCT group ( P<0.05). Conclusions:Compared with the peripheral blood stem cell transplantation group, the umbilical cord blood transplantation patients had a faster rate of lymphocyte reconstitution, and patients received umbilical cord blood transplantation had a stronger ability of immune reconstitution and could achieve long-term hematopoiesis.
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BACKGROUND: In recent years, umbilical cord blood has gradually become a crucial alternative source of stem cells for related and unrelated bone marrow or peripheral blood hematopoietic stem cell transplantation, which is increasingly used in the treatment of hematological malignancies in children. OBJECTIVE: To compare the clinical efficacy of sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation for treating hematological malignancies in children. METHODS: The clinical data of children with hematological malignancies who received umbilical cord blood transplantation at the Hematopoietic Stem Cell Transplantation Center of the First Affiliated Hospital of Zhengzhou University between January 1, 1998 and December 31, 2018 was retrospectively analyzed. All the patients received myelablative conditioning regimen, and cyslosporine A combined with or without mycophenolate mofetil were concurrently adopted for graft-versus-host disease prophylaxis. RESULTS AND CONCLUSION: (1) Two patients in the sibling donor umbilical cord blood transplantation group and three in the unrelated umbilical cord blood transplantation group did not attain hematological engraftment and subsequently died from infection, and other patients succeeded in hematological engraftment. The median time of neutrophil and platelet engraftment in the sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups was [17 days (11-43 days), 18 days (12-45 days), P=0.307] and [20.5 days (15-50 days), 27 days (18-56 days), P=0.773]. There was no significant difference between the two groups. (2) The incidence of acute graft-versus-host disease and chronic graft-versus-host disease in the sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups was 36% vs. 43% (P=0.737) and 15% vs. 33% (P=0.412). There was no significant difference between the two groups. There was also no significant difference in the incidence of infection after transplantation between sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups (56% vs. 71%, P=0.343). (3) There were no significant differences in the 2-year overall survival (61% vs. 36%, P=0.301), or 2-year relapse-free survival (56% vs. 33%, P=0.151). The 5-year overall survival and 5-year relapse-free survival in the sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups were 54% vs. 24% (P=0.044) and 50% vs. 20% (P=0.039). The results showed that there was a significant difference in long-term survival rate between two groups. (4) Our results reveal that both sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation are safe, effective and applicable for children with hematological malignancies. In particular, there are significant benefits in the long-term survival of substitute donor transplantation for pediatric patients with hematological malignancies.
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Hematopoietic stem cell transplantation is a pivotal method to treat hematological malignancies, hematopoietic failure diseases and inherited metabolic diseases. Umbilical cord blood is one of the main sources of hematopoietic stem cells and the most ideal cell source for stem cell research and application. In this article, the clinical application of umbilical cord blood transplantation for more than 30 years was reviewed from the aspects of the history, current situation, advantages, improvement, application prospect and expectation of umbilical cord blood transplantation.
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Objective To explore the curative effect and prognosis of umbilical cord blood in the treatment of hematological diseases in children. Method The clinical data of 51 children who underwent umbilical cord blood transplantation from January 2011 to June 2016 were analyzed retrospectively. Results In 51 children (34 males and 17 females) with median age of 62 months, 32 children had malignant hematologic diseases and 19 children had nonmalignant hematologic diseases. Two children died before the granulocytes were reconstructed, 4 children had primary implantation failure, and 45 children had successfully implantation. The median time of implantation was 16 d, and the median time of platelet implantation was 23 d. The incidence of peri-implantation syndrome was 46.94%. The 100 day survival rate and long-term overall survival (OS) in children with peri-implantation syndrome were (73.9±9.2)% and (50.2±11.7)% respectively, which were significantly lower than the OS (100%) in children without peri-implantation syndrome (P<0.01). The incidence of acute graft versus host disease (aGVHD) was 55.10%, among which Ⅱ-Ⅲ degrees of aGVHD was 28.57% and Ⅳdegrees of aGVHD was 26.53%. The 100 day OS in children with Ⅳ degrees of aGVHD was (61.5±13.5)%, and The OS in children with Ⅲ and Ⅳ degrees of aGVHD were (75.0±21.7)% and (44.9±14.1)% respectively, and the OS in children without aGVHD was (90.2±6.6)%. The difference was statistically significant (χ2=14.35,P=0.002). The incidence of chronic GVHD (cGVHD) was 28.57%. The long-term OS in children with cGVHD was (72.7±13.4)%, while OS in children without cGVHD was 100%. The 100 days OS was (86.0±4.9)%. Long-term OS in cord blood transplantation was (77.9±6.3)%, among which OS for malignant hematological diseases was (76.6±7.8)% and OS for nonmalignant hematological diseases was (79.5±11.3)%. Among malignant hematological diseases, the OS in acute lymphoblastic leukemia (ALL) was (87.5±11.7)%, OS in acute myeloid lymphocytic leukemia (AML) was (76.7±10.3)%, and OS in myelodysplastic syndrome (MDS) was (33.3±27.2)%. Conclusions Umbilical cord blood transplantation is an effective treatment for hematologic diseases in children. It is important to treat the peri-implantation syndrome. Prevention and treatment Ⅲ/Ⅳ degree of aGVHD and cGVHD are important strategies to improve the efficacy of umbilical cord blood transplantation.
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Objective To explore the curative effect and prognosis of umbilical cord blood in the treatment of hematological diseases in children. Method The clinical data of 51 children who underwent umbilical cord blood transplantation from January 2011 to June 2016 were analyzed retrospectively. Results In 51 children (34 males and 17 females) with median age of 62 months, 32 children had malignant hematologic diseases and 19 children had nonmalignant hematologic diseases. Two children died before the granulocytes were reconstructed, 4 children had primary implantation failure, and 45 children had successfully implantation. The median time of implantation was 16 d, and the median time of platelet implantation was 23 d. The incidence of peri-implantation syndrome was 46.94%. The 100 day survival rate and long-term overall survival (OS) in children with peri-implantation syndrome were (73.9±9.2)% and (50.2±11.7)% respectively, which were significantly lower than the OS (100%) in children without peri-implantation syndrome (P<0.01). The incidence of acute graft versus host disease (aGVHD) was 55.10%, among which Ⅱ-Ⅲ degrees of aGVHD was 28.57% and Ⅳdegrees of aGVHD was 26.53%. The 100 day OS in children with Ⅳ degrees of aGVHD was (61.5±13.5)%, and The OS in children with Ⅲ and Ⅳ degrees of aGVHD were (75.0±21.7)% and (44.9±14.1)% respectively, and the OS in children without aGVHD was (90.2±6.6)%. The difference was statistically significant (χ2=14.35,P=0.002). The incidence of chronic GVHD (cGVHD) was 28.57%. The long-term OS in children with cGVHD was (72.7±13.4)%, while OS in children without cGVHD was 100%. The 100 days OS was (86.0±4.9)%. Long-term OS in cord blood transplantation was (77.9±6.3)%, among which OS for malignant hematological diseases was (76.6±7.8)% and OS for nonmalignant hematological diseases was (79.5±11.3)%. Among malignant hematological diseases, the OS in acute lymphoblastic leukemia (ALL) was (87.5±11.7)%, OS in acute myeloid lymphocytic leukemia (AML) was (76.7±10.3)%, and OS in myelodysplastic syndrome (MDS) was (33.3±27.2)%. Conclusions Umbilical cord blood transplantation is an effective treatment for hematologic diseases in children. It is important to treat the peri-implantation syndrome. Prevention and treatment Ⅲ/Ⅳ degree of aGVHD and cGVHD are important strategies to improve the efficacy of umbilical cord blood transplantation.
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Rare diseases are very rare,but usually have severe symptoms.Some rare diseases are life-threatening.Most rare diseases cannot be cured.A very small part of these diseases can be cured by hematopoietic stem cell transplantation (HSCT).Umbilical cord blood transplantation(UCBT) is more suitable for children for the weak T cell immunity,the lower request for human leukocyte antigen (HLA) identity type and the lower incidence of graft versus host disease(GVHD).This article reviewed the published data in the treatment of UCBT in primary immunodeficiency disease,inherited metabolic disease,inflammatory bowel disease and bone marrow failure syndrome,in order to improve the level of rare disease treatment by HSCT,especially for UCBT.
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Treatments for patients with hematologic malignancies not in remission are limited, but a few clinical studies have investigated the effects of salvaged unrelated cord blood transplantation (CBT). We retrospectively studied 19 patients with acute leukemia, 5 with myelodysplastic syndrome (MDS with refractory anemia with excess blasts [RAEB]), and 2 with non-Hodgkin's lymphoma who received 1 CBT unit ≤2 loci human leukocyte antigen (HLA)-mismatched after undergoing myeloablative conditioning regimens between July 2005 and July 2014. All of them were in non-remission before transplantation. The infused total nucleated cell (TNC) dose was 4.07 (range 2.76-6.02)×107/kg and that of CD34+ stem cells was 2.08 (range 0.99-8.65)×105/kg. All patients were engrafted with neutrophils that exceeded 0.5×109/L on median day +17 (range 14-37 days) and had platelet counts of >20×109/L on median day +35 (range 17-70 days). Sixteen patients (61.5%) experienced pre-engraftment syndrome (PES), and six (23.1%) patients progressed to acute graft-versus-host disease (GVHD). The cumulative incidence rates of II-IV acute GVHD and chronic GVHD were 50% and 26.9%, respectively. After a median follow-up of 27 months (range 5-74), 14 patients survived and 3 relapsed. The estimated 2-year overall survival (OS), disease-free survival (DFS), and non-relapse mortality (NRM) rates were 50.5%, 40.3%, and 35.2%, respectively. Salvaged CBT might be a promising modality for treating hematologic malignancies, even in patients with a high leukemia burden.
Subject(s)
Adolescent , Adult , Child , Female , Humans , Male , Young Adult , Allografts , Anemia, Refractory, with Excess of Blasts/therapy , Cord Blood Stem Cell Transplantation , Graft vs Host Disease , Leukemia, Biphenotypic, Acute/therapy , Lymphoma, Non-Hodgkin/therapy , Anemia, Refractory, with Excess of Blasts/mortality , Cord Blood Stem Cell Transplantation/mortality , Disease-Free Survival , Follow-Up Studies , Graft vs Host Disease/mortality , Hematologic Neoplasms/mortality , Hematologic Neoplasms/therapy , Leukemia, Biphenotypic, Acute/mortality , Leukemia, Lymphoid/mortality , Leukemia, Lymphoid/therapy , Leukemia, Myeloid/mortality , Leukemia, Myeloid/therapy , Leukemia/mortality , Leukemia/therapy , Lymphoma, Non-Hodgkin/mortality , Myelodysplastic Syndromes/mortality , Myelodysplastic Syndromes/therapy , Retrospective Studies , Remission Induction/methods , Treatment OutcomeABSTRACT
PURPOSE: Preterm very low birth weight infant have high rate of adverse neurodevelopmental sequale. Recently, there have been lots of reports that human umbilical cord blood transplantation ameliorates functional deficits in animal models as hypoxic ischemic injury. This pilot study was undertaken to determine the clinical efficacy and safety of autologous umbilical cord blood cell transplantation for preventing neurodevelopmental sequale in perterm VLBW. METHODS: Subjects were 26 preterm infants whose birth weight are less than 1,500 g and delivered under the intrauterine period 34 weeks. Autologous umbilical mononuclear cells (about 5.87x10(7)/kg) were injected to neonate via the umbilical vein on the postnatal 24-48 hour. The therapeutic efficacy was assessed by numbers of nucleated RBC, urinary uric acid/creatinine ratio, concentration of neuron specific enolase (NSE), interleukin 6 (IL6), interleukin-1beta (IL-1beta), and glial cell derived neurotrophic factor (GDNF) in serum and cerebrospinal fluid on day 1 and 7. RESULTS: There were no significant differences in the numbers of the nucleated RBC, urinary uric acid/creatinine ratio, concentration of creatine kinase between the transplanted infants and controls. But the nucleated RBC is more likely to be rapidly discharged in the transplanted group. In the transplanted group, the concentrations of IL6, IL-1beta, and GDNF were no significant difference between day 1 and 7, although GDNF seemed to be elevated. Serum NSE concentration was significantly elevated after transplantation, but not in CSF. CONCLUSION: It is suggested that autologous umbilical cord blood transplantation in preterm very low birth weight infant is safe to apply clinical practice. Long term follow up study should be needed to evaluate the potential therapeutic effect of umbilical cord blood transplantation for neuroprotection.