Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies

Heredia, Cristina Díaz de; Ortega, Juan José López; Díaz, Miguel Ángel; Olivé, T; Badell, Isabel; González‐Vicent, Marta; Toledo, Josep Sánchez de

doi:10.1038/sj.bmt.1705946

Cited by 29 publications

(30 citation statements)

References 43 publications

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“…The different genetic background of the transplanted units did not seem to adversely affect the outcome in our patients; our engraftment rate is 93% and our 100-TRM is 15%, conforming to already published data. 1,[3][4][5][6]15,16 In the malignant disorder category, we report in this study an estimated OS and EFS of 41 and 38%, respectively, and others also have reported similar figures. 7,9,15 The degree of HLA disparity seemed to have an effect on the survival of patients with non-malignant hematological disorders, as has been shown by Gluckman et al 3 in Fanconi anemia (FA) patients, although others failed to show such an effect in patients with severe aplastic anemia.…”

Section: Second Transplantssupporting

confidence: 71%

“…We report in this study a relatively large number of patients (32 patients), 17 of whom are long-term disease-free survivors; the reported survival rates in the literature are between 73 and 86%. 4,5,17 One health measure required by the ministry of health for all neonates in Saudi Arabia is the routine administration of the Bacille Calmette-Gue´rin vaccine after birth, and hence in the absence of a PID family history, many of these children are referred for allo-SCT and are critically ill with disseminated Tuberculosis after receiving this vaccine. The use of RIC may be particularly reasonable in this subset of patients because of the co-morbid conditions, but the available data on RIC in cord blood transplantation are, although encouraging, mostly based on single-center studies and are limited by the small number of patients and the short follow-up.…”

Section: Discussionmentioning

confidence: 99%

“…[1][2][3][4][5][6][7][8] In UCB transplants, the degree of HLA matching has been closely associated with long-term survival. Thus, it is now a major goal of public cord blood banking to provide a large and diverse inventory of HLA typing to extend transplant access to racial and ethnic minorities.…”

Section: Introductionmentioning

confidence: 99%

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Unrelated cord blood transplantation in pediatric patients: a report from Saudi Arabia

Ayas

Al-Seraihi

Al‐Jefri

et al. 2009

Bone Marrow Transplant

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In unrelated cord blood (UCB) transplantation, survival has been shown to correlate with the degree of HLA matching. Thus, to extend transplant access to different ethnic backgrounds, many western UCB transplantation banks now encourage donation from non-Caucasians. Until recently, Saudi Arabia did not have a national UCB bank. In this study we report our experience in UCB transplantation in children using units procured from western cord blood banks. A total of 97 children underwent unrelated UCB transplantation at King Faisal Specialist Hospital and Research Center (KFSHRC), of which 95 were of Arab ethnicity. A total of 30 patients had malignant disorders, 25 patients had non-malignant hematological disorders and 42 patients had inborn errors. Conditioning was according to disease, with six patients receiving reduced-intensity regimens. In all, 46 patients received one-Ag-mismatched units and 51 received twoAg-mismatched units. Engraftment occurred in 93% of patients, the 100-TRM was 15%, acute GVHD developed in 20% of patients and chronic GVHD occurred in 9% of patients. The 5-year OS and EFS estimates were 52 and 43%, respectively. The search for UCB transplantation units for Saudi patients in western banks yielded reasonably compatible units for our patients; the results are consistent with published data. Our data are encouraging for UCB transplant programs in countries in which there are no national UCB transplantation banks.

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Section: Second Transplantssupporting

confidence: 71%

Section: Discussionmentioning

confidence: 99%

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Unrelated cord blood transplantation in pediatric patients: a report from Saudi Arabia

Ayas

Al-Seraihi

Al‐Jefri

et al. 2009

Bone Marrow Transplant

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“…1 The best results have been obtained with HLA-identical sibling transplants. 2 Promising results have been reported using haplo-identical donors in children with a SCID, although immune reconstitution after such transplants is often delayed for 3-6 months. 3 For patients with other types of immune deficiency, an unrelated BM transplant remains the only potential therapeutic option.…”

Section: Introductionmentioning

confidence: 94%

“…Several case reports and single-institution studies have reported on the use of unrelated CBT in children with an immune deficiency. 2,[7][8][9][10] Those studies use different preparative regimens and GVHD prophylaxis, which makes the results difficult to interpret. The Cord Blood Transplantation Study (COBLT) is a prospective study sponsored by the National Institute of Health to evaluate the feasibility and safety of CBT in children and adults with malignant and nonmalignant diseases.…”

Section: Introductionmentioning

confidence: 99%

Unrelated umbilical cord blood transplantation in children with immune deficiency: results of a multicenter study

Frangoul

Wang

Harrell

et al. 2009

Bone Marrow Transplant

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In the absence of a related donor, unrelated cord blood transplant (CBT) may be a potential option for patients with a primary immune deficiency (PID). Most published experience consists of single-center data using multiple preparative regimens and GVHD prophylaxis. We report the results of a multicenter prospective trial of unrelated CBT for PID. A total of 24 children with PID, with a median age of 1 year (range: 0.23-7.81 years) and a median weight of 10.5 kg (range: 4-24.4 kg) received unrelated CBT between 1999 and 2003. All patients received a fully ablative conditioning regimen with identical GVHD prophylaxis and supportive care. Most patients (79%) received a 1 or 2 HLA Ag-mismatched cord unit with a median nucleated cell infused of 9.3 Â 10 7 /kg (range: 1.0-31.2) and a median CD34 of 2.7 Â 10 5 /kg 2.9 (range: 0.6-84.5). The cumulative incidence of neutrophil engraftment by day 42 was 58% (95% CI: 38-79%) at a median of 19 days. Cumulative incidence estimates of grade III-IV acute GVHD at day 100 and chronic GVHD at 1 year were 29% (95% CI: 10-48%) and 24% (95% CI: 3-44%), respectively. The probability of survival at 180 days and 1 year was 66.7% (95% CI: 44.3-81.7%) and 62.5% (95% CI: 40.3-78.4%), respectively. Unrelated CBT should be considered in children with PID.

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Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott–Aldrich syndrome

Yan

Shi

Song

et al. 2018

Pediatric Blood & Cancer

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Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies

Cited by 29 publications

References 43 publications

Unrelated cord blood transplantation in pediatric patients: a report from Saudi Arabia

Unrelated cord blood transplantation in pediatric patients: a report from Saudi Arabia

Unrelated umbilical cord blood transplantation in children with immune deficiency: results of a multicenter study

Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott–Aldrich syndrome

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