Unrelated umbilical cord stem cell transplantation for X-linked immunodeficiencies

Ziegner, Ulrike; Ochs, Hans D.; Schanen, Carolyn; Feig, Stephen A.; Seyama, Kuniaki; Futatani, Takeshi; Gross, Thomas G.; Wakim, Mary; Roberts, Robert L.; Rawlings, David J.; Dovat, Sinisa; Fraser, John K.; Stiehm, E. Richard

doi:10.1067/mpd.2001.112511

Cited by 45 publications

(25 citation statements)

References 11 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…If available, transplantation from either an HLA-matched sibling or an HLA-matched unrelated donor can be performed safely. Umbilical cord blood may be an alternative for those without an HLA-matched donor [22]. GVHD prophylaxis with cyclosporine and short-term methotrexate is feasible, although one should be aware of thrombotic microangiopathy, one of the complications induced by cyclosporine.…”

Section: Discussionmentioning

confidence: 99%

Allogeneic hematopoietic stem cell transplantation for seven children with X‐linked hyper‐IgM syndrome: A single center experience

et al. 2004

View full text Add to dashboard Cite

X-linked hyper-IgM syndrome (XHIM), or hyper-IgM syndrome type 1 (HIGM1), is a rare primary immunodeficiency disorder susceptible to recurrent bacterial infection and opportunistic infection such as Pneumocystis carinii and Cryptosporidium parvum. The longterm outcome is quite poor, and allogeneic hematopoietic stem cell transplantation (HSCT) offers the only cure. Seven patients with XHIM, from age 3 to 19 years (mean 11.3 years), underwent allogeneic HSCT in our institution. Details of pre-and post-transplantation data and transplantation procedure were analyzed retrospectively. The donors were HLA-identical siblings for three patients and HLA-identical unrelated donors for four patients. All but one received conventional conditioning regimen consisting of busulfan and cyclophosphamide and prophylaxis for graft-versus-host disease (GVHD) consisting of cyclosporine and methotrexate. Five out of seven patients are alive and well with normal CD40L expression, and four of these five are free of intravenous immunoglobulin supplementation. The two patients who died had prolonged episodes of severe and recurrent infections and organ damage. We conclude that conventional allogeneic HSCT from HLA matched related or unrelated donors is curative and feasible for XHIM patients, if performed before significant infections and organ damage occur. For the high-risk patients, an alternative approach including nonmyeloablative HSCT may be more feasible. Am.

show abstract

Section: Discussionmentioning

confidence: 99%

Allogeneic hematopoietic stem cell transplantation for seven children with X‐linked hyper‐IgM syndrome: A single center experience

et al. 2004

View full text Add to dashboard Cite

show abstract

“…Up to 2005, 14 patients had been transplanted from different types of donors, with overall survival of 71%. The first case of a related UCB transplant was published in 1993 25 and the first two cases of UCB unrelated transplants in 2001; 21 all were successful. The first of our two patients had a brother who died from Epstein-Barr Virus-associated Bcell lymphoma.…”

Section: Discussionmentioning

confidence: 99%

“…These advantages are notable in PID, particularly SCID. Apart from sporadic reported cases of UCB transplants in PID, [21][22][23][24][25][26][27] only data from three centres have been published. 30,31,32 In the St Louis series, six of eight patients were cured and one remained alive with chronic interstitial pneumonitis.…”

Section: Discussionmentioning

confidence: 99%

“…[17][18][19][20] Since then, some cases of PID successfully treated with UCB transplant have been described. [21][22][23][24][25][26][27] The Eurocord group reported the preliminary results in 47 children from 25 centres with 69% survival at 2 years. 18,28,29 Experiences of single centres in St Louis, Newcastle and Tokyo were recently published.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies

Heredia

Ortega

Díaz

et al. 2007

Bone Marrow Transplant

View full text Add to dashboard Cite

“…In this case, conditioning consisted of busulfan (BU), cyclophosphamide (CY), etoposide (VP-16) and anti-thymocyte globulin (ATG). 8 We report a successful URCBT for a CD40LD patient with the conditioning of BU+CY and investigated the immunologic reconstitution during the period of URCBT.…”

mentioning

confidence: 99%

Successful unrelated cord blood transplantation for a patient with CD40 ligand deficiency

Tsuji¹,

Imai²,

Morinishi³

et al. 2007

Haematologica

View full text Add to dashboard Cite

show abstract

Unrelated umbilical cord stem cell transplantation for X-linked immunodeficiencies

Cited by 45 publications

References 11 publications

Allogeneic hematopoietic stem cell transplantation for seven children with X‐linked hyper‐IgM syndrome: A single center experience

Allogeneic hematopoietic stem cell transplantation for seven children with X‐linked hyper‐IgM syndrome: A single center experience

Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies

Successful unrelated cord blood transplantation for a patient with CD40 ligand deficiency

Contact Info

Product

Resources

About