Nonmyeloablative allogeneic bone marrow transplantation of a child with systemic autoimmune disease and lung vasculitis

Jones, Olcay Y.; Cahill, Richard A.

doi:10.1007/s12026-007-0015-1

Cited by 3 publications

(4 citation statements)

References 36 publications

(32 reference statements)

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“…Initially fully myeloablative protocols shifted towards less toxic RIC regimens, often resulting in mixed hematopoietic engraftment that may be adding to the induction of tolerance by the postulated 'graft-versus-autoimmunity' effect [15,16 && ,74,75]. Concerns about high TRM and risk from GvHD are the main reasons for a small number of adult patients with rheumatic diseases undergoing allo-HSCT, and only very few children have been reported [13,15,[76][77][78][79][80][81][82]. However, two retrospective outcome analyses of allo-HSCT in 50 patients with severe autoimmune diseases, of which 16 with rheumatic diseases, reported surprisingly good results.…”

Section: Allogeneic Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

“…Table 2(a) summarises the outcome of allo-HSCT for 28 patients with severe paediatric rheumatic diseases [13,[76][77][78][79][80][81][82]85,86]. Of the 18 patients with JIA, 15 improved within the first year post-HSCT and 13 of 15 (86.6%) achieved remission, whilst disease relapsed within 6 months post-HSCT in one of the two patients with only partial improvement.…”

Section: Allogeneic Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

“…Two patients from this cohort of severe JIA transplanted as ‘the last resort’ treatment died, resulting in TRM of 11.7% [85,86] (P. Veys, A. Lazareva, M. Slatter, P. Sedlacek, S. Chandra, R. Marsh, personal communications). Reassuringly there were no deaths reported in a smaller cohort of 10 children transplanted for other paediatric rheumatic diseases at the ‘salvage state’; long-term CR was achieved in six (60%), PR in three, and one disease relapse occurred with a graft loss [13,76–82] (M. Slatter, T. Cole, D. Hughes, personal communications). Details of unpublished patients are shown in Supplementary Table 3, http://links.lww.com/COR/A50.…”

Section: Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

“…Alternatively, as engraftment exclusively in the Treg cell compartment was sufficient to cure IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked) syndrome, an IEI with severely impaired function of T-reg cells due to FOXP3 mutation, this may also apply for autoimmune diseases [91–93]. The effect of nonhaematopoietic mesenchymal stromal cells (MSC) and the survival of posttransplant minimal residual autoimmune disease have also been suggested to play a role [79,80,89,90 ▪▪ ,94].…”

Section: Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

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Haematopoietic stem cell transplantation in paediatric rheumatic disease

Abinun

Slatter

2021

Current Opinion in Rheumatology

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Purpose of reviewA small proportion of children affected by rheumatic diseases suffer from severe, progressive disease, resistant to conventional antirheumatic therapies and to biologic agents interfering with inflammatory cytokines, costimulatory molecules expressed on immune system cells and intracellular signalling pathways. Adding to the poor prognosis is a high risk from significant morbidity and mortality associated with longterm treatment with multiple, often combined anti-inflammatory and immunosuppressive agents. Carefully selected patients from this unfortunate group may benefit from treatment with haematopoietic stem cell transplantation. Recent findingsThe majority of patients with severe paediatric rheumatic and autoinflammatory diseases treated with autologous and/or allogeneic haematopoietic stem cell transplantation achieved long-term remission. However, the incidence of disease relapse and transplant related morbidity and mortality is still significant. SummaryCareful patient and donor selection, timing of the transplant earlier in the course of disease rather than the 'last resort' and choosing the most suitable conditioning regimen for each individual patient are the major factors favouring successful outcome. Close co-operation between the patients, their family, and involved medical teams is essential. Keywords auto-inflammatory diseases, complete remission, disease relapse, haematopoietic stem cell transplantation, paediatric rheumatic diseases, transplant-related mortality && ]. For such children, the only treatment option is haematopoietic stem cell transplantation (HSCT), often referred to as the 'last resort' or 'salvage' therapy [12,13].

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Section: Allogeneic Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

Section: Allogeneic Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

Section: Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

Section: Haematopoietic Stem Cell Transplantationmentioning

confidence: 99%

See 2 more Smart Citations

Haematopoietic stem cell transplantation in paediatric rheumatic disease

Abinun

Slatter

2021

Current Opinion in Rheumatology

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Hematopoietic stem cell transplantation for pediatric autoimmune disease: where we stand and where we need to go

Krauss

Kamani

2009

Bone Marrow Transplant

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Emerging concepts in haematopoietic cell transplantation

Sykes

2012

Nat Rev Immunol

110

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Haematopoietic cell transplantation (HCT) is the most widely used cellular therapy. It is the only known cure for some hematologic malignancies and has recently been used for additional indications, such as allograft tolerance induction and treatment of autoimmune diseases. Recent advances have enabled HCT in a wider range of patients with improved outcomes. This Review summarizes the latest developments in this therapy, focusing on issues that will impact future advancement.

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Nonmyeloablative allogeneic bone marrow transplantation of a child with systemic autoimmune disease and lung vasculitis

Cited by 3 publications

References 36 publications

Haematopoietic stem cell transplantation in paediatric rheumatic disease

Haematopoietic stem cell transplantation in paediatric rheumatic disease

Hematopoietic stem cell transplantation for pediatric autoimmune disease: where we stand and where we need to go

Emerging concepts in haematopoietic cell transplantation

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