Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood.

نویسندگان

  • Christian Flotho
  • Brigitte Strahm
  • Udo Kontny
  • Ulrich Duffner
  • Anke M J Peters
  • Wolfgang Dupuis
  • Charlotte M Niemeyer
چکیده

Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic disorder characterized by chronic or intermittent intravascular haemolysis, variable cytopenia and an increased risk of thrombosis. Stem cell transplantation (SCT) is a curative therapeutic option, but its risks must be carefully weighed against the natural course of PNH. World-wide experience with SCT for PNH in the paediatric age group is scarce. We report on two adolescents suffering from PNH with life-threatening complications who were successfully transplanted from unrelated donors. Indications and techniques of SCT in childhood PNH are discussed and an overview of the literature is given.

برای دانلود متن کامل این مقاله و بیش از 32 میلیون مقاله دیگر ابتدا ثبت نام کنید

ثبت نام

اگر عضو سایت هستید لطفا وارد حساب کاربری خود شوید

منابع مشابه

Paroxysmal nocturnal haemoglobinuria and Budd-Chiari syndrome.

An 11 year old boy developed pancytopenia, haemolysis, and Budd-Chiari syndrome. The venous thrombosis extended to involve other intra-abdominal vessels before paroxysmal nocturnal haemoglobinuria was recognised as the underlying haematological abnormality. Earlier diagnosis would have made curative bone marrow transplantation a possibility.

متن کامل

Renal transplantation for end-stage renal disease due to paroxysmal nocturnal haemoglobinuria.

Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by nocturnal haemoglobinuria, haemolytic anaemia, and thrombosis [ 1]. Most patients have anatomical and functional renal abnormalities, although renal function deterioration often remains unrecognized. The pathogenesis could be related to iron deposition or to repeated vascular thrombosis. The present case illustrates that renal trans...

متن کامل

Haemoglobinuria And Portal Venous Thrombosis In A Young Male.

Paroxysmal nocturnal haemoglobinuria is a non-malignant stem cell disorder due to acquired somatic mutations in cell surface anchored proteins CD55 and CD59. Both have a compliment inhibitory role and their deficiency leads to intravascular haemolysis. This paper reports a challenging case of a 25 years old male who presented with generalized weakness, exertional dyspnoea and episodic early mor...

متن کامل

Paroxysmal nocturnal haemoglobinuria: nature's gene therapy?

The development of paroxysmal nocturnal haemoglobinuria (PNH) requires two coincident factors: somatic mutation of the PIG-A gene in one or more haemopoietic stem cells and an abnormal, hypoplastic bone marrow environment. When both of these conditions are met, the fledgling PNH clone may flourish. This review will discuss the pathophysiology of this disease, which has recently been elucidated ...

متن کامل

Paroxysmal nocturnal haemoglobinuria at Oslo University Hospital 2000-2010.

BACKGROUND Paroxysmal nocturnal haemoglobinuria (PNH) is a rare haematological disease characterised by chronic haemolysis, pancytopenia and venous thrombosis. The condition is attributable to a lack of control of complement attack on erythrocytes, thrombocytes and leukocytes, and can be diagnosed by means of flow cytometry. In this quality assurance study, we have reviewed information from the...

متن کامل

ذخیره در منابع من


  با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید

برای دانلود متن کامل این مقاله و بیش از 32 میلیون مقاله دیگر ابتدا ثبت نام کنید

ثبت نام

اگر عضو سایت هستید لطفا وارد حساب کاربری خود شوید

عنوان ژورنال:
  • British journal of haematology

دوره 118 1  شماره 

صفحات  -

تاریخ انتشار 2002