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Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy

Chiesa, Robert, Boelens, Jaap Jan, Duncan, Christine N., Kühl, Jörn-Sven, Sevin, Caroline, Kapoor, Neena, Prasad, Vinod K., Lindemans, Caroline A., Jones, Simon A., Amartino, Hernan M., Algeri, Mattia, Bunin, Nancy, Diaz-de-Heredia, Cristina, Loes, Daniel J., Shamir, Esther, Timm, Alison, McNeil, Elizabeth, Dietz, Andrew C., Orchard, Paul J.·2022·Blood Advances
DOI 10.1182/bloodadvances.2021005294PMC PMC8905699
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Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) in early cerebral adrenoleukodystrophy can stabilize neurologic function and improve survival but has associated risks including transplant-related mortality (TRM), graft failure, and graft-versus-host disease (GVHD). An observational study of 59 patients with median age at allo-HSCT of 8 years addressed impact of donor source, donor match, conditioning regimen, and cerebral disease stage on first allo-HSCT outcomes. Efficacy analyses included 53 patients stratified by disease category: advanced disease (AD; n = 16) with Loes score >9 or neurological function score (NFS) >1 and 2 early disease (ED) cohorts (ED1 [Loes ≤4 and NFS ≤1; n = 24] and ED2 [Loes >4-9 and NFS ≤1; n = 13]). Survival free of major functional disabilities and without second allo-HSCT at 4 years was significantly higher in the ED (66%) vs AD (41%) cohort (P = .015) and comparable between ED1 and ED2 cohorts (P = .991). The stabilization of neurologic function posttransplant was greater in the ED vs AD cohort, with a median change from baseline at 24 months after allo-HSCT in NFS and Loes score, respectively, of 0 and 0.5 in ED1 (n = 13), 0.5 and 0 in ED2 (n = 6), and 2.5 and 3.0 (n = 4) in AD cohort. TRM was lower in the ED (7%) compared with the AD (22%) cohort; however, the difference was not significant (P = .094). Transplant-related safety outcomes were also affected by transplant-related characteristics: graft failure incidence was significantly higher with unrelated umbilical cord grafts vs matched related donors (P = .039), and acute GVHD and graft failure incidences varied by conditioning regimen. This study was registered at www://clinicaltrials.gov as #NCT02204904.

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Crossref · 10.1182/bloodadvances.2021005294

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Graphical abstract Visual AbstractCC BY-NC-ND · PubMed Central
Figure 1. Kaplan-Meier analyses of overall survival (A) MFD-free survival and (B) by disease severity cohorts. Time of origin was the first allo-HSCT and patients alive without an event were censored at the last follow-up or at the time of study termination. MFD-free survival included survival without second allo-HSCT or major functional disabilities (MFDs).
Figure 1. Kaplan-Meier analyses of overall survival (A) MFD-free survival and (B) by disease severity cohorts. Time of origin was the first allo-HSCT and patients alive without an event were censored at the last follow-up or at the time of study termination. MFD-free survival included survival without second allo-HSCT or major functional disabilities (MFDs).CC BY-NC-ND · PubMed Central
Figure 2. Loes score and Neurological Function Score (NFS) over time. Median change from baseline by disease stage is shown for Loes score (A) and NFS (B). Number of evaluable patients at each time point are shown below the x-axis (data availability for each patient at each visit was influenced by a number of factors, including the survival status, retransplantation status, length of follow-up, whether the visit took place, and whether the assessment was performed). (C-D) Individual patient Loes scores and NFS by disease stage.
Figure 2. Loes score and Neurological Function Score (NFS) over time. Median change from baseline by disease stage is shown for Loes score (A) and NFS (B). Number of evaluable patients at each time point are shown below the x-axis (data availability for each patient at each visit was influenced by a number of factors, including the survival status, retransplantation status, length of follow-up, whether the visit took place, and whether the assessment was performed). (C-D) Individual patient Loes scores and NFS by disease stage.CC BY-NC-ND · PubMed Central
Figure 3. Gadolinium enhancement (GdE) status over time. Results for individual patients are stratified by disease severity. Patients with graft failure/rejection or chimerism <90% are indicated.
Figure 3. Gadolinium enhancement (GdE) status over time. Results for individual patients are stratified by disease severity. Patients with graft failure/rejection or chimerism <90% are indicated.CC BY-NC-ND · PubMed Central

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Citations

Cites

Show all 7 references

Research works printed but not linked: 26

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Metadata

Authors
Chiesa, R., Boelens, J. J., Duncan, C. N., Kühl, J.-S., Sevin, C., Kapoor, N., Prasad, V. K., Lindemans, C. A., Jones, S. A., Amartino, H. M., Algeri, M., Bunin, N., Diaz-de-Heredia, C., Loes, D. J., Shamir, E., Timm, A., McNeil, E., Dietz, A. C., & Orchard, P. J.
Affiliations
  • Bone Marrow Transplant Department, Great Ormond Street Hospital for Children, London, UK;
  • Stem Cell Transplantation andCellular Therapies, Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY;
  • Department of Pediatric Oncology, Boston Children's Hospital and Dana-Farber Cancer Institute, Boston, MA;
  • Stem Cell Transplantation, Department of Pediatric Oncology, Hematology and Hemostasis, University Hospital Leipzig, Leipzig, Germany;
  • Pediatric Neurology Department, Leukodystrophies Reference Center, Hôpital Universitaire Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Paris, France;
  • Hematology, Oncology and Blood & Marrow Transplantation, Department of Pediatrics, Children's Hospital Los Angeles, Los Angeles, CA;
  • Division of Pediatric Transplant and Cellular Therapy, Department of Pediatrics, Duke University Medical Center, Durham, NC;
  • Pediatric Blood and Bone Marrow Transplantation Program, Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands;
  • Paediatric Inborn Errors of Metabolism, Manchester University NHS Foundation Trust, Manchester, UK;
  • Child Neurology Service, Hospital Universitario Austral, Buenos Aires, Argentina;
  • Department ofPediatric Hematology and Oncology, Cell and Gene Therapy,Bambino GesùChildren's Hospital,IRRCS, Rome, Italy;
  • Cellular Therapy and Transplant Section, Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA;
  • Paediatric Oncology and Haematology Department, Hospital Universitari Vall d'Hebron and Vall d'Hebron Institut de Recerca, Barcelona, Spain;
  • Midwest Radiology, Minneapolis, MN;
  • bluebird bio, Inc., Cambridge, MA; and
  • Blood and Marrow Transplantation Program, Department of Pediatrics, University of Minnesota
ORCID
Venue
Blood Advances
Publisher
American Society of Hematology
Published
2022-03-03
Volume
6
Issue
5
Pages
1512-1524
ISSN
  • 2473-9529
  • 2473-9537
DOI
10.1182/bloodadvances.2021005294
PMC
PMC8905699
Cited by
40
References
7
Language
en

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CoChat. (2026). Citation check: Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy. https://app.cochat.ai/papers/10.1182/bloodadvances.2021005294
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Chiesa, R., Boelens, J. J., Duncan, C. N., Kühl, J.-S., Sevin, C., Kapoor, N., Prasad, V. K., Lindemans, C. A., Jones, S. A., Amartino, H. M., Algeri, M., Bunin, N., Diaz-de-Heredia, C., Loes, D. J., Shamir, E., Timm, A., McNeil, E., Dietz, A. C., & Orchard, P. J. (2022). Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy. Blood Advances. https://doi.org/10.1182/bloodadvances.2021005294