Toggle Main Menu Toggle Search

Open Access padlockePrints

Allogeneic hematopoietic stem cell transplantation in leukocyte adhesion deficiency type I and III

Lookup NU author(s): Dr Mary Slatter, Professor Andrew GenneryORCiD


Full text for this publication is not currently held within this repository. Alternative links are provided below where available.


© 2021 by The American Society of Hematology.Type I and III leukocyte adhesion deficiencies (LADs) are primary immunodeficiency disorders resulting in early death due to infections and additional bleeding tendency in LAD-III. The curative treatment of LAD-I and LAD-III is allogeneic hematopoietic stem cell transplantation (allo-HSCT). In this retrospective multicenter study, data were collected using the European Society for Blood and Marrow Transplantation registry; we analyzed data from 84 LAD patients from 33 centers, all receiving an allo-HSCT from 2007 to 2017. The 3-year overall survival estimate (95% confidence interval [CI]) was 83% (74-92) for the entire cohort: 84% (75-94) and 75% (50-100) for LAD-I and LAD-III, respectively. We observed cumulative incidences (95% CI) of graft failure (GF) at 3 years of 17% (9%-26%) and grade II to IV acute graft-versus-host disease (aGVHD) at 100 days of 24% (15%-34%). The estimate (95% CI) at 3 years for GF- and GVHD-II to IV-free survival as event-free survival (EFS) was 56% (46-69) for the entire cohort; 58% (46-72) and 56% (23-88) for LAD-I and LAD-III, respectively. Grade II to IV acute GVHD was a relevant risk factor for death (hazard ratio 3.6; 95% CI 1.4-9.1; P 5.006). Patients' age at transplant $13 months, transplantation from a nonsibling donor, and any serological cytomegalovirus mismatch in donor-recipient pairs were significantly associated with severe acute GVHD and inferior EFS. The choice of busulfan- or treosulfan-based conditioning, type of GVHD prophylaxis, and serotherapy did not impact overall survival, EFS, or aGVHD. An intrinsic inflammatory component of LAD may contribute to inflammatory complications during allo-HSCT, thus providing the rationale for considering anti-inflammatory therapy pretreatment.

Publication metadata

Author(s): Bakhtiar S, Salzmann-Manrique E, Blok H-J, Eikema D-J, Hazelaar S, Ayas M, Toren A, Goldstein G, Moshous D, Locatelli F, Merli P, Michel G, Ozturk G, Schulz A, Heilmann C, Ifversen M, Wynn RF, Aleinikova O, Bertrand Y, Tbakhi A, Veys P, Karakukcu M, Kupesiz A, Ghavamzadeh A, Handgretinger R, Unal E, Perez-Martinez A, Gokce M, Porta F, Aksu T, Karasu G, Badell I, Ljungman P, Skorobogatova E, Yesilipek A, Zuckerman T, Bredius RRG, Stepensky P, Shadur B, Slatter M, Gennery AR, Albert MH, Bader P, Lankester A

Publication type: Article

Publication status: Published

Journal: Blood Advances

Year: 2021

Volume: 5

Issue: 1

Pages: 262-273

Print publication date: 12/01/2021

Online publication date: 11/01/2021

Acceptance date: 31/10/2020

ISSN (print): 2473-9529

ISSN (electronic): 2473-9537

Publisher: American Society of Hematology


DOI: 10.1182/bloodadvances.2020002185


Altmetrics provided by Altmetric