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Long-term remission in pediatric Wegener granulomatosis following allo-SCT after reduced-intensity conditioning

Bone Marrow Transplantation volume 46, pages 462–463 (2011)Cite this article

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Wegener granulomatosis (WG) is a multisystemic necrotizing granulomatous vasculitis of small- and medium-sized vessels, primarily involving the respiratory tract and the kidneys. Involvement of other organ systems such as eye, subglottis, joints, skin, nervous system and liver has been reported. Pediatric WG is rare, shares many features with the adult disease, but shows a predominance of female patients, a higher incidence of subglottic stenosis and of generalized disease. In total, 80–95% of pediatric patients are positive for classical anti-neutrophil cytoplasmic antibodies (ANCA) and 5–20% are positive for perinuclear (P)-ANCA. By treatment with steroids and CY, a remission rate of 75% can be achieved. However, the relapse rate is high (about 75%) and significant chronic organ damage is observed. In children, a survival rate between 85 and 100% is reported.1, 2, 3

Marmont and VanBekkum were the first to discuss the potential role of allo-SCT for refractory autoimmune diseases.4 Here, we report the successful treatment of a pediatric WG by allo-SCT following reduced-intensity conditioning.

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Acknowledgements

We thank Professor Gerhard Fritsch, Professor Winfried Pickl and Professor Gerhard Zlabinger for providing data about immunoreconstitution.

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  1. St Anna Children's Hospital, SCT-Unit, Vienna, Austria

    A Lawitschka, C Peters, M G Seidel, A Havranek, T Fazekas, E D Gueclue, H Gadner & S Matthes-Martin

  2. Children's Cancer Research Institute (CCRI), Vienna, Austria

    A Heitger

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  1. A Lawitschka
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  2. C Peters
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  3. M G Seidel
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  7. E D Gueclue
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Correspondence to A Lawitschka.

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Lawitschka, A., Peters, C., Seidel, M. et al. Long-term remission in pediatric Wegener granulomatosis following allo-SCT after reduced-intensity conditioning. Bone Marrow Transplant 46, 462–463 (2011). https://doi.org/10.1038/bmt.2010.126

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