Clinical Outcomes of Umbilical Cord Blood Transplantation Using Ex Vivo Expansion: A Systematic Review and Meta-Analysis of Controlled Studies

Saiyin, Tana; Kirkham, Aidan M.; Bailey, Adrian; Shorr, Risa; Pineault, Nicolas; Maganti, Harinad; Allan, David S.

doi:10.1016/j.jtct.2022.11.007

Cited by 10 publications

(7 citation statements)

References 27 publications

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“…While an RCT was completed [ 23 ], it did not enroll sufficiently to detect a difference in the primary outcome of progression-free survival, but did observe improved overall survival with haploidentical bone marrow transplant compared to double cord blood transplantation using reduced-intensity regimens. With favorable results from cord blood expansion studies [ 24 ], more studies will be needed to clarify the precise role of cord blood transplantation in adults and children.…”

Section: Discussionmentioning

confidence: 99%

A Portrait of Cord Blood Units Distributed for Transplantation from Canadian Blood Services’ Cord Blood Bank: First Analysis

et al. 2022

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Background: The Canadian Blood Services Cord Blood Bank (CBS CBB) was created to improve access to stem cell products for transplantation for patients across ethnic groups. An analysis of distributed units is needed to assess the effectiveness of the bank to meet the needs of patients from different ethnic groups. Methods: A descriptive analysis was performed on all cord blood units distributed from the CBS’ CBB as of 30 June 2022. Results: Distribution of the first 60 units based on CBS’ CBB inventory has been linear over time. A similar proportion of cord blood unit (CBU) recipients were pediatric or adult. More than half of the cord blood units (56.7%) were distributed to recipients outside of Canada, and CBUs were used to treat a broad range of hematologic and immune disorders. 43.3% of distributed CBUs were of non-Caucasian ethnicity and 18% were from donors self-reporting as multi-ethnic. The mean total nucleated cell counts and total CD34+ cell counts were 1.9 ± 0.1 × 109 cells and 5.3 ± 0.5 × 106 CD34+ cells, respectively. CD34+ cells per kg (recipient weight) varied significantly between pediatric (age 0–4), adolescent (age 5–17) and adult recipients (age 18 and older) (3.1 ± 0.5, 1.4 ± 0.5 and 0.9 ± 0.07 × 105 CD34+ cells/kg, respectively). HLA matching was 6/6 (15%), 5/6 (47%) or 4/6 (38%). Conclusions: The CBS’ CBB has facilitated the utilization of banked units for patients across a broad range of ages, geographic distribution, ethnicity, and diseases. Distributed units were well matched for HLA alleles and contained robust cell counts, reflecting a high-quality inventory with significant utility.

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Section: Discussionmentioning

confidence: 99%

A Portrait of Cord Blood Units Distributed for Transplantation from Canadian Blood Services’ Cord Blood Bank: First Analysis

et al. 2022

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“…Allogeneic hematopoietic cell transplantation is the sole potential curative therapy option in many hematological malignancies (Lin et al 2023 ). Hematopoietic stem cells can be collected from umbilical cord blood as well as bone marrow and peripheral blood (Saiyin et al 2023 ). The disadvantage of umbilical cord blood is that it provides low numbers of hematopoietic stem cells, which may lead to treatment failure.…”

Section: Hematologymentioning

confidence: 99%

“…The disadvantage of umbilical cord blood is that it provides low numbers of hematopoietic stem cells, which may lead to treatment failure. An ex vivo culture strategy has been developed to enhance the number of hematopoietic stem cells (Saiyin et al 2023 ). Omidubicel-onlv is a nicotinamide-modified stem cell transplant that is derived from the blood of the umbilical cord (Heo 2023a ).…”

Section: Hematologymentioning

confidence: 99%

A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2023

Kayki-Mutlu,

Aksoyalp,

Wojnowski

et al. 2024

Naunyn-Schmiedeberg's Arch Pharmacol

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With 54 new drugs and seven cellular and gene therapy products, the approvals by the US Food and Drug Administration (FDA) recovered 2023 from the 2022 dent back to the levels of 2020–2021. As in previous years of this annual review, we assign these new drugs to one of three levels of innovation: first drug against a condition (“first-in-indication”), first drug using a novel molecular mechanism (“first-in-class”), and “next-in-class,” i.e., a drug using an already exploited molecular mechanism. We identify four (7%) “first-in-indication,” 22 (36%) “first-in-class,” and 35 (57%) “next-in-class” drugs. By treatment area, rare diseases (54%) and cancer drugs (23%) were once again the most prevalent (and partly overlapping) therapeutic areas. Other continuing trends were the use of accelerated regulatory approval pathways and the reliance on biopharmaceuticals (biologics). 2023 marks the approval of a first therapy based on CRISPR/Cas9 gene editing.

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“…Another strategy to improve CB expansion is to add certain agents to the culture media ( 156 ). A variety of molecules, including Notch ligand ( 157 , 158 ), copper chelation agents such as TEPA ( 159 ), vitamin B derivatives such as nicotinamide ( 160 ), aryl hydrocarbon receptor antagonists (AHRs) ( 161 ), and pyrimidoindole derivative UM171 ( 162 ), have shown improved expansion in clinical studies ( Table 3 ).…”

Section: Cb-derived Stem Cell Therapymentioning

confidence: 99%

Umbilical cord blood derived cellular therapy: advances in clinical development

Wang

Metheny

2023

Front. Oncol.

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While cord blood (CB) is primarily utilized in allogeneic hematopoietic cell transplantation (HCT), the development of novel cell therapy products from CB is a growing and developing field. Compared to adult blood, CB is characterized by a higher percentage of hematopoietic stem cells (HSCs) and progenitor cells, less mature immune cells that retain a high capacity of proliferation, and stronger immune tolerance that requires less stringent HLA-matching when used in the allogenic setting. Given that CB is an FDA regulated product and along with its unique cellular composition, CB lends itself as a readily available and safe starting material for the development of off-the-shelf cell therapies. Moreover, non-hematologic cells such as mesenchymal stem cell (MSCs) residing in CB or CB tissue also have potential in regenerative medicine and inflammatory and autoimmune conditions. In this review, we will focus on recent clinical development on CB-derived cellular therapies in the field of oncology, including T-cell therapies such as chimeric antigen receptor (CAR) T-cells, regulatory T-cells, and virus-specific T-cells; NK-cell therapies, such as NK cell engagers and CAR NK-cells; CB-HCT and various modifications; as well as applications of MSCs in HCT.

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Clinical Outcomes of Umbilical Cord Blood Transplantation Using Ex Vivo Expansion: A Systematic Review and Meta-Analysis of Controlled Studies

Cited by 10 publications

References 27 publications

A Portrait of Cord Blood Units Distributed for Transplantation from Canadian Blood Services’ Cord Blood Bank: First Analysis

A Portrait of Cord Blood Units Distributed for Transplantation from Canadian Blood Services’ Cord Blood Bank: First Analysis

A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2023

Umbilical cord blood derived cellular therapy: advances in clinical development

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