Studies | EBMT

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Study type

Disease

Group

Type of transplant / Cell therapy

Text

Setting the stage for newborn screening and gene therapy: a disease-specific retrospective survey of FHL2 and 3

Études rétrospectives

Troubles héréditaires

Inborn Errors Working Party (IEWP)

Type de traitement

Investigateur principal

Stephan Ehl, Despina Moshous, Claire Booth, Sujal Ghosh

Outcome of haploidentical transplant in chronic granulomatous disease

Études rétrospectives

Troubles héréditaires

Inborn Errors Working Party (IEWP)

Type de traitement

Allogénique - Haplo-identique

Investigateur principal

Benedicte Neven

Late effects after hematopoietic stem cell transplantation in patients with HLH

Retrospective Studies

Other non-malignant disorders

Paediatric Diseases Working Party (PDWP)

Inborn Errors Working Party (IEWP)

Transplant Complications Working Party (TCWP)

Type de traitement

Investigateur principal

AnnaCarin Horne

The outcome of second and third allogeneic conditioned HSCT in patients with non-SCID primary immune deficiency (PID) following primary or secondary graft failure

Retrospective Studies

Inborn Errors Working Party (IEWP)

Type de traitement

Investigateur principal

A prospective outcome study on patients with profound combined immune deficiency (p-CID)

Retrospective Studies

Inborn Errors Working Party (IEWP)

Type de traitement

Investigateur principal

Outcomes of HLA-mismatched HSCT in patients with PID following in vitro T-cell depletion with CD3+TCRαβ/CD19 depleted PBSC or in vivo T-cell depleted HSCT with post-transplant cyclophosphamide

Retrospective Studies

Other non-malignant disorders

Inborn Errors Working Party (IEWP)

Type de traitement

Allogeneic - Haploidentical

Investigateur principal

Mary Slatter & Michael Albert

Immunodeficiency – centromeric instability – facial dysmorphism (ICF) syndrome: evaluation of characteristics and outcome after allogeneic haematopoietic stem cell transplantation

Retrospective Studies

Inborn Errors Working Party (IEWP)

Type de traitement

Investigateur principal

Dagmar Berghuis

HSCT experience in patients with Interferon gamma receptor deficiencies

Retrospective Studies

Inborn Errors Working Party (IEWP)

Type de traitement

Investigateur principal