Transplantation of highly purified peripheral-blood CD34+ progenitor cells from related and unrelated donors in children with nonmalignant diseases

Lang, Peter; Klingebiel, Thomas; Bader, Peter; Greil, Johann; Schumm, Michael; Schlegel, Paul-Gerhard; Eyrich, Matthias; Mueller-Weihrich, S; Niethammer, D.; Handgretinger, Rupert

doi:10.1038/sj.bmt.1704303

Cited by 34 publications

(30 citation statements)

References 41 publications

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“…In contrast to this, we used the concept of megadoses of CD34 þ selected cells, which resulted in lower GvHD rates and quick engraftment even in the haploidentical setting. 27,28 Kremens et al 29 reported on six children with nonmalignant diseases who received a CD34 þ selected graft from a parental donor. No GvHD 4II, no transplant-related mortality, and timely engraftment were reported in this series.…”

Section: Discussionmentioning

confidence: 99%

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler)

Grigull

Beilken

Schrappe

et al. 2004

Bone Marrow Transplant

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Summary:Hurler syndrome (MPS1H) is a progressive inborn error of mucopolysaccharide metabolism leading to premature death. Allogeneic hematopoietic cell transplantation (HCT) can achieve stabilization and improve long-term survival. However, large studies have shown that preparative regimen-related toxicity (RRT) and graft failure rates have been relatively high. We transplanted five Hurler children with a fludarabine-based conditioning regimen, consisting of fludarabine/busulphan/ATG for matched family donor (MFD), with the addition of melphalan for mismatched family donor and matched unrelated donor (MUD) transplantations. Median age at HCT was 27 months (range 10-36). The source of stem cells was bone marrow in one MFD and CD34-selected PBSC in four patients. Median CD34 þ cell dose was 25 Â 10 6 /kg (range 11.5-54). No RRT 4grade II was observed. All patients are surviving at a median of 32 months (range 14-41) and show sustained donor engraftment with 3/5 having full donor chimerism, and 2/5 mixed chimerism (485%). We conclude that this regimen is feasible and has low toxicity in Hurler children. In combination with high doses of CD34 þ selected cells (410 Â 10 6 /kg) and donor lymphocyte infusions, stable engraftment could be achieved in unrelated and mismatched related transplantations. Bone Marrow Transplantation (2005) 35, 265-269.

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Section: Discussionmentioning

confidence: 99%

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler)

Grigull

Beilken

Schrappe

et al. 2004

Bone Marrow Transplant

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“…DLI in the HLA-mismatched setting carries a significant risk for acute GVHD. 29,30 The major complications of transplant were viral reactivation and PTLD, with 1 late transplant-related mortality. PTLD occurred more frequently than would be expected from a T replete allogeneic transplant 31 and from a CD34 1 cell-selected graft.…”

Section: Discussionmentioning

confidence: 99%

Alternative donor hematopoietic stem cell transplantation for sickle cell disease

Gilman

Eckrich

Epstein³

et al. 2017

Blood Advances

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“…Although a final assessment about the use of haploidentical stem cell transplantation based on these results is not possible so far, it is tempting to speculate that the megadose concept will result in superior engraftment rates with a concomitant low incidence of acute and chronic GvHD. In fact, Lang et al reported a series of 25 patients with nonmalignant diseases (excluding FA) where transplantation of large numbers of positively enriched CD34 ϩ hematopoietic stem cells from related or unrelated donors resulted in a survival rate of 88% without acute GvHD greater than grade II and limited chronic GvHD in only 8% of the patients [12]. Future studies have to show whether these encouraging results will also apply to FA patients.…”

Section: Transplantation Beyond Hla-barriersmentioning

confidence: 99%

Stem Cell Transplantation in Fanconi Anemia – Recent Advances with Alternative Donors

Eyrich¹,

Winkler²,

Schlegel³

2007

Fanconi Anemia

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Bone marrow transplantation from an HLA-identical sibling donor is the treatment of choice for Fanconi anemia (FA) patients with bone marrow failure. However, with today's small size families less than 25% of FA patients have a matching sibling donor. The remaining patients can be treated with stem cell transplantations from alternative donors such as matched unrelated or haploidentical donors. While results with conventional bone marrow transplantation continually improved, outcome after alternative donor transplantation remained poor due to high rates of transplant-related complications, graft rejection and graft-versus-host disease (GvHD). Recently, advances with less myeloablative and more immunosuppressive conditioning regimens have demonstrated promising results. Insights into the mechanisms of immune reconstitution could show that at least in children immune recovery after haploidentical stem cell transplantation is fast and results in complete restoration of normal immune function within the first year after transplantation. Finally, innovations in the field of stem cell collection and processing have led to cellular graft compositions which now provide reliable engraftment in almost all patients with concomitant low incidence of GvHD. This review discusses these recent advances in alternative donor transplantations and concludes that this treatment option should be an early recommendation for FA patients with bone marrow failure who lack an HLA-identical sibling donor.Although the name of the disease suggests a purely hematological disorder, Fanconi anemia (FA) is a systemic disease which is characterized by physical abnormalities, progressive bone marrow failure and increased risk of malignancies. A typical diagnostic feature, which might in part explain some of these symptoms, is increased chromosome breakage at baseline, and even more

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Transplantation of highly purified peripheral-blood CD34+ progenitor cells from related and unrelated donors in children with nonmalignant diseases

Cited by 34 publications

References 41 publications

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler)

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler)

Alternative donor hematopoietic stem cell transplantation for sickle cell disease

Stem Cell Transplantation in Fanconi Anemia – Recent Advances with Alternative Donors

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