Outcomes of allogeneic haematopoietic stem cell transplantation from HLA-matched and alternative donors: a European Society for Blood and Marrow Transplantation registry retrospective analysis

Shouval, Roni; Fein, Joshua; Labopin, Myriam; Kröger, Nicolaus; Duarte, Rafael F.; Bader, Peter; Chabannon, Christian; Kuball, Jürgen; Basak, Grzegorz W.; Dufour, Carlo; Galimard, Jacques-Emmanuel; Polge, Emmanuelle; Lankester, Arjan C.; Montoto, Silvia; Snowden, John A.; Styczyński, Jan; Yakoub‐Agha, Ibrahim; Mohty, Mohamad; Nagler, Arnon

doi:10.1016/s2352-3026(19)30158-9

Cited by 181 publications

(116 citation statements)

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“…Hematopoietic-cell transplantation (HCT) is an established procedure for many inherited or acquired disorders of the hematopoietic system, whether benign or neoplastic, including those of the immune system, and as enzyme replacement in metabolic disorders [1][2][3]. The activity survey of the European Society of Blood and Marrow Transplantation (EBMT), describing the status of HCT in Europe and affiliated countries, has become an instrument to observe trends and to monitor changes in the technology in Europe and neighboring countries [4][5][6][7][8][9][10][11][12][13][14]. The survey using a standardized structure captures the numbers of HCT from highly committed participating teams, divided by indication, donor type, and stem cell source.…”

Section: Introductionmentioning

confidence: 99%

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

Passweg¹,

Baldomero²,

Chabannon³

et al. 2020

Bone Marrow Transplant

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164

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Hematopoietic-cell transplantation (HCT) is widely used for acquired and congenital disorders of the hematopoietic system. Number of transplants performed in Europe and associated countries continues to rise with 47,468 HCT in 42,901 patients [19,630 allogeneic (41%) and 27,838 autologous (59%)] reported by 701 centers in 50 countries in 2018. Main indications were myeloid malignancies 10,679 (25%; 97% allogeneic), lymphoid malignancies 27,318 (64%; 20% allogeneic), solid tumors 1625 (4%; 2.9% allogeneic), and nonmalignant disorders 3063 (7%; 81% allogeneic). This year's analysis focuses on cellular therapies with the marked growth in CAR T-cell therapies from 151 in 2017 to 301 patients reported in 2018. Other cellular therapy numbers show less significant changes. Important trends in HCT include a 49% increase in allogeneic HCT for chronic phase CML (although transplant numbers remain low) and a 24% increase in aplastic anemia. In autologous HCT, there is an ongoing increase in autoimmune diseases (by 19%), predominantly due to activity in multiple sclerosis. This annual report reflects current activity and highlights important trends, useful for health care planning.

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Section: Introductionmentioning

confidence: 99%

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

Passweg¹,

Baldomero²,

Chabannon³

et al. 2020

Bone Marrow Transplant

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164

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“…Currently, haploidentical donors have been an alternative source for allo-stem cell transplantation (SCT) for patients that require transplantation but have no related or unrelated donors with matching human leukocyte antigen (HLA) [1][2][3][4][5]. With the increasingly used haploidentical SCT (HIDT), HLA-identical sibling donors remain the first choice, though a number of studies have shown that treating patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) using haploidentical donors (HIDs) could achieve comparable outcomes to those who undergoing HLA-matched sibling donor transplantation (MSDT) [3,6,7].…”

Section: Introductionmentioning

confidence: 99%

“…With the increasingly used haploidentical SCT (HIDT), HLA-identical sibling donors remain the first choice, though a number of studies have shown that treating patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) using haploidentical donors (HIDs) could achieve comparable outcomes to those who undergoing HLA-matched sibling donor transplantation (MSDT) [3,6,7]. On the other hand, using haploidentical transplants, the graft-versus-leukemia (GVL) effect may be stronger, as mismatches for HLA antigens on leukemic cells would provide allo-immune targets [4,5,[8][9][10][11][12][13]. A recent large European Society for Blood and Marrow Transplantation (EBMT) study indicated that HIDT has a lower incidence of relapse than MSDT for low-risk (HR = 0.83, P = 0.011) and intermediate-risk (HR = 0.85, P = 0.033) hematological malignancies [5].…”

Section: Introductionmentioning

confidence: 99%

“…On the other hand, using haploidentical transplants, the graft-versus-leukemia (GVL) effect may be stronger, as mismatches for HLA antigens on leukemic cells would provide allo-immune targets [4,5,[8][9][10][11][12][13]. A recent large European Society for Blood and Marrow Transplantation (EBMT) study indicated that HIDT has a lower incidence of relapse than MSDT for low-risk (HR = 0.83, P = 0.011) and intermediate-risk (HR = 0.85, P = 0.033) hematological malignancies [5]. The better relapse rate with HIDT compared to MSDT has also been observed in patients with lymphomas [8,9].…”

Section: Introductionmentioning

confidence: 99%

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Haploidentical donor is preferred over matched sibling donor for pre-transplantation MRD positive ALL: a phase 3 genetically randomized study

Chang¹,

Wang

et al. 2020

J Hematol Oncol

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Background: Previous reports suggest a benefit associated with haploidentical donor transplantation (HIDT) compared to matched sibling donor transplantation (MSDT) in certain contexts, and the choice of optimal candidates warrants further investigation. Methods: We designed a prospective genetically randomized study to evaluate donor options between acute lymphoblastic leukemia (ALL) patients positive for measurable residual disease (MRD) pre-transplantation who underwent HIDT (n = 169) or MSDT (n = 39). Results: The cumulative incidence of positive MRD post-transplantation was 26% (95% CI, 19-33%) and 44% (95% CI, 28-60%) for HIDT and MSDT, respectively (P = 0.043). Compared to the HIDT cohort, the MSDT cohort had a higher 3-year cumulative incidence of relapse (CIR; 47%, 95% CI, 31-63% vs. 23%, 95% CI, 17-29%; P = 0.006) and lower 3-year probability of leukemia-free survival (LFS; 43%, 95% CI, 27-59% vs. 65%, 95% CI, 58-72%; P = 0.023) and overall survival (OS; 46%, 95% CI, 30-62% vs. 68%, 95% CI, 61-75%; P = 0.039), without a difference in non-relapse-mortality (10%, 95% CI, 1-19% vs. 11%, 95% CI, 6-16%; P = 0.845). Multivariate analysis showed that HIDT is associated with a low CIR (HR = 0.364; 95% CI, 0.202-0.655; P = 0.001) and better LFS (HR = 0.414; 95% CI, 0.246-0.695; P = 0.001) and OS (HR = 0.380; 95% CI, 0.220-0.656; P = 0.001). Conclusions: HIDT is better than MSDT in view of favorable anti-leukemia activity for patients with pre-transplantation MRD positive ALL. The current study paves the way to determine that haploidentical donors are the preferred choice regardless of available matched sibling donors in a subgroup population.

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“…The HLA genes are the most polymorphic in the human genome encoding more than 25 000 allelic variants . The characterization and matching of HLA alleles between donor and recipient is important for the successful outcome of hematopoietic stem cell and solid organ transplantation …”

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confidence: 99%

Characterization of the novel HLA‐DPA1 allele, HLA‐DPA101:27*

Ingrassia

Pecoraro

Blando

et al. 2020

HLA

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Outcomes of allogeneic haematopoietic stem cell transplantation from HLA-matched and alternative donors: a European Society for Blood and Marrow Transplantation registry retrospective analysis

Cited by 181 publications

References 31 publications

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

Haploidentical donor is preferred over matched sibling donor for pre-transplantation MRD positive ALL: a phase 3 genetically randomized study

Characterization of the novel HLA‐DPA1 allele, HLA‐DPA101:27*

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Outcomes of allogeneic haematopoietic stem cell transplantation from HLA-matched and alternative donors: a European Society for Blood and Marrow Transplantation registry retrospective analysis

Cited by 181 publications

References 31 publications

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

Haploidentical donor is preferred over matched sibling donor for pre-transplantation MRD positive ALL: a phase 3 genetically randomized study

Characterization of the novel HLA‐DPA1 allele, HLA‐DPA1*01:27

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Characterization of the novel HLA‐DPA1 allele, HLA‐DPA101:27*