scholarly journals Umbilical Cord Blood: An Evolving Stem Cell Source for Sickle Cell Disease Transplants

2013 ◽  
Vol 2 (5) ◽  
pp. 337-340 ◽  
Author(s):  
Shalini Shenoy
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Cord Blood ◽  
Umbilical Cord ◽  
Sickle Cell ◽  
Umbilical Cord Blood ◽  
Cell Disease ◽  
Stem Cell Source ◽  
Cell Source

scholarly journals Umbilical Cord Blood Transplantation for Children with Thalassemia and Sickle Cell Disease

2011 ◽  
Vol 17 (9) ◽  
pp. 1375-1382 ◽  
Author(s):  
Annalisa Ruggeri ◽  
Mary Eapen ◽  
Andromachi Scaravadou ◽  
Mitchell S. Cairo ◽  
Monica Bhatia ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cord Blood ◽  
Umbilical Cord ◽  
Sickle Cell ◽  
Umbilical Cord Blood ◽  
Cord Blood Transplantation ◽  
Umbilical Cord Blood Transplantation ◽  
Cell Disease ◽  
Blood Transplantation

scholarly journals Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients

2017 ◽  
Vol 28 (3) ◽  
pp. 136-144 ◽  
Author(s):  
Naoya Uchida ◽  
Atsushi Fujita ◽  
Matthew M. Hsieh ◽  
Aylin C. Bonifacino ◽  
Allen E. Krouse ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Bone Marrow ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
Stem Cell Source ◽  
Cell Source

Hematopoietic Stem Cell Transplantation for Sickle Cell Disease in Childhood: A Single Center Experience with 45 Patients

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3103-3103 ◽  
Author(s):  
Laurence Dedeken ◽  
Phu-Quoc Le ◽  
Nadira Azzi ◽  
Cecile Brachet ◽  
Catherine Heijmans ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cord Blood ◽  
Sickle Cell ◽  
Cell Disease ◽  
Hematopoietic Stem

Abstract Abstract 3103 Despite improvement in medical management, sickle cell disease (SCD) is still associated with high risk of morbidity, chronic disability and early death. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative approach. Since November 1988, 45 patients (median age: 8.3 years; range: 1.7–15.3 years) with severe SCD underwent related HSCT in our unit. Thirty-five received bone marrow transplant, 3 cord blood, 6 bone marrow and cord blood and 1 peripheral blood stem cells. Two donors result from preimplantation genetic diagnosis with HLA selection. All were HLA-identical sibling except one who had one class II mismatch. All had one or more severe manifestations: 24 patients presented more than 2 vaso-occlusive crises per year, 11 recurrent acute chest syndrome, 19 cerebral vasculopathy and 4 erythroid alloimmunisation. Conditioning regimen consisted of the standard combination of busulfan, cyclophosphamide and from November 1991 antithymocyte globulins (ATG) were added: ATG Fresenius first and from July 2000 ATG Merieux. Since 1995 all patients were treated with hydroxyurea (HU) prior to transplantation for a median duration of 2.7 years (range: 0.8–10.7 years). Acute graft versus host disease (GVHD) was observed in 11 patients (3 grade III and 2 grade IV). Ten patients were treated for CMV reactivation and 4 for EBV reactivation. Only one patient had presented a probable invasive fungal disease. After median follow-up of 6.5 years, 10 patients had presented chronic GVHD, none was extensive. Only one required therapy beyond 2 years from transplant. Engraftment was successful in 42/45. One rejection occurred 15 months after transplantation. Since HU introduction before transplant (1995), no graft failure occurred. Important mixed chimerism is present in 2 patients (AA donor) who remain free of any sickle cell disease symptoms. Two deaths occurred: 1 unexplained death 6 years after HSCT in a child free of any treatment and 1 cerebral hemorrhage 18 days after transplant in a child with severe cerebral vasculopathy. Growth was normal after transplant. As expected, gonadal function was impaired in the majority of girls. However 3 girls had spontaneous normal puberty and one had two spontaneous pregnancies with normal outcome. Our results are very encouraging showing excellent outcomes. Both the overall survival (OS: 95.6%) and the event-free survival (EFS: 86.7%) are comparable to the other published studies, ranging from 93 to 97%, and 82 to 86 % respectively. Since 1995, all the 33 patients engrafted successfully. Previous treatment with HU may have contributed to successful engraftment. After 5.3 years of follow-up, their OS and EFS are both at 96.9%. The difference in outcome before and after 1995 is strongly significant for EFS (58.3% vs 96.9%, p=0.003). Severe cerebral vasculopathy with its risk of CNS hemorrhage remains a true challenge. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Allogeneic stem cell transplantation with omidubicel in sickle cell disease

2021 ◽  
Vol 5 (3) ◽  
pp. 843-852
Author(s):  
Suhag Parikh ◽  
Joel A. Brochstein ◽  
Einat Galamidi ◽  
Aurélie Schwarzbach ◽  
Joanne Kurtzberg
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Stem Cell Transplantation ◽  
Cord Blood ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Ex Vivo ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
Cell Dose

Abstract Many patients with sickle cell disease (SCD) do not have HLA-matched related donors for hematopoietic stem cell transplantation (HSCT). Unrelated cord blood (UCB) is an alternative graft option but is historically associated with high graft failure rates, with inadequate cell dose a major limitation. Omidubicel is a nicotinamide-based, ex vivo–expanded UCB product associated with rapid engraftment in adults with hematologic malignancies. We hypothesized that increasing the UCB cell dose with this strategy would lead to improved engraftment in pediatric patients undergoing myeloablative HSCT for SCD. We report the outcomes of a phase 1/2 study in 13 patients with severe SCD who received omidubicel in combination with an unmanipulated UCB graft and 3 who received a single omidubicel graft. Grafts were minimally matched with patients at 4 of 6 HLA alleles. Median age at transplant was 13 years. A median CD34+ expansion of ∼80-fold was observed in omidubicel and led to rapid neutrophil engraftment (median, 7 days). Long-term engraftment was derived from the unmanipulated graft in most of the double cord blood recipients. Two of the 3 single omidubicel recipients also had sustained engraftment. Incidence of acute graft-versus-host disease (GVHD) was high, but resolved in all surviving patients. Event-free survival in the double cord group was 85% (median follow-up 4 years). All 3 patients in the single cord group were alive at 1 year after transplantation. Ex vivo expansion of UCB with omidubicel supports engraftment in patients with SCD. This approach to decreasing the incidence of GVHD should be optimized for general use in patients with SCD. This study was registered at www.clinicaltrials.gov as #NCT01590628.

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