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Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis
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| Zeitschriftentitel: | Blood |
|---|---|
| Personen und Körperschaften: | , , , , , , , , , , , , |
| In: | Blood, 112, 2008, 11, S. 4338-4338 |
| Format: | E-Article |
| Sprache: | Englisch |
| veröffentlicht: |
American Society of Hematology
|
| Schlagwörter: |
| author_facet |
Argiolu, Francesca Sanna, Maria Adele Bartoli, Antonella Ferrara, Rocco Piroddi, Antonio Addari, Carmen Orofino, Maria Grazia Rizzo, Fabiana Cossu, Fausto Scalas, Maria Rosa Broglia, Monica Giagu, Nicolina Galanello, Renzo Argiolu, Francesca Sanna, Maria Adele Bartoli, Antonella Ferrara, Rocco Piroddi, Antonio Addari, Carmen Orofino, Maria Grazia Rizzo, Fabiana Cossu, Fausto Scalas, Maria Rosa Broglia, Monica Giagu, Nicolina Galanello, Renzo |
|---|---|
| author |
Argiolu, Francesca Sanna, Maria Adele Bartoli, Antonella Ferrara, Rocco Piroddi, Antonio Addari, Carmen Orofino, Maria Grazia Rizzo, Fabiana Cossu, Fausto Scalas, Maria Rosa Broglia, Monica Giagu, Nicolina Galanello, Renzo |
| spellingShingle |
Argiolu, Francesca Sanna, Maria Adele Bartoli, Antonella Ferrara, Rocco Piroddi, Antonio Addari, Carmen Orofino, Maria Grazia Rizzo, Fabiana Cossu, Fausto Scalas, Maria Rosa Broglia, Monica Giagu, Nicolina Galanello, Renzo Blood Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis Cell Biology Hematology Immunology Biochemistry |
| author_sort |
argiolu, francesca |
| spelling |
Argiolu, Francesca Sanna, Maria Adele Bartoli, Antonella Ferrara, Rocco Piroddi, Antonio Addari, Carmen Orofino, Maria Grazia Rizzo, Fabiana Cossu, Fausto Scalas, Maria Rosa Broglia, Monica Giagu, Nicolina Galanello, Renzo 0006-4971 1528-0020 American Society of Hematology Cell Biology Hematology Immunology Biochemistry http://dx.doi.org/10.1182/blood.v112.11.4338.4338 <jats:title>Abstract</jats:title> <jats:p>Until now few patients with renal failure on hemodialysis have undergone haematopoietic stem-cell transplantation (HSCT), and none with thalassaemia. Case report. Patient of 45 years, UPN 168, with β Thalassaemia major, genetic compound β0 39 C--&gt;T/β0 6-A, non transfusible due to red blood cell immunization (positive DAT, Hb 5–7 g/dl), under hemodialytic treatment for three years for bilateral focal nephrosclerosis. He suffered from cardiopathy (atrial thrombosis, pulmonary hypertension, atrial and ventricular dilatation), hypothyroidism, hepatopathy (HCV positive with grade II haemosiderosis). Quality of life was much harmed by the severe chronic anaemia and nephropathy. In December 2007 we performed HSCT from his HLA identical brother. Conditioning regimen included: Busulfan (Bu) IV (Busilvex Pierre Fabre Médicament) in single daily dose (3.5 mg/kg/day) for 4 days, Cyclophosphamide 40 mg/kg/day for 3 days. GvHD prophylaxis consisted of Cyclosporin 3 mg/kg/day IV (days -2 to +1), 2 mg/kg/day IV (days +2 to +42), changed to oral administration of 4 mg/kg/day from day +43. Hemodialysis was performed every other day in sterile room. Nucleated marrow cells infused: 4.4 × 108/kg. A mixed chimerism (VNTR more than 90% donor) was documented at day +19. ANC &gt;0.5 × 109/L at day +21. PLT &gt;20 × 109/L at day +15. Eight months after transplantation, patient is in good general condition, with stable mixed chimerism (more than 95% donor), haemoglobin above 11 g/dl, leucocytes and platelets in normal range. Substantial improvement of chronic anaemia ameliorated patient’s quality of life and now patient can be considered a kidney transplant candidate. This single case report does not allow definitive conclusions to be drawn regarding HSCT suitability of patients with chronic renal failure on hemodialysis. We can observe that once-daily intravenous busulfan administration (after the hemodialysis session) has made it possible to keep therapeutic range, as has been ascertained by pharmacokinetic study of busulfan.</jats:p> Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis Blood |
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| title |
Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
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Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_full |
Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_fullStr |
Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_full_unstemmed |
Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_short |
Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_sort |
haematopoietic stem cell transplantation in thalassaemic patient with renal failure on hemodialysis |
| topic |
Cell Biology Hematology Immunology Biochemistry |
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http://dx.doi.org/10.1182/blood.v112.11.4338.4338 |
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2008 |
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4338-4338 |
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<jats:title>Abstract</jats:title>
<jats:p>Until now few patients with renal failure on hemodialysis have undergone haematopoietic stem-cell transplantation (HSCT), and none with thalassaemia. Case report. Patient of 45 years, UPN 168, with β Thalassaemia major, genetic compound β0 39 C--&gt;T/β0 6-A, non transfusible due to red blood cell immunization (positive DAT, Hb 5–7 g/dl), under hemodialytic treatment for three years for bilateral focal nephrosclerosis. He suffered from cardiopathy (atrial thrombosis, pulmonary hypertension, atrial and ventricular dilatation), hypothyroidism, hepatopathy (HCV positive with grade II haemosiderosis). Quality of life was much harmed by the severe chronic anaemia and nephropathy. In December 2007 we performed HSCT from his HLA identical brother. Conditioning regimen included: Busulfan (Bu) IV (Busilvex Pierre Fabre Médicament) in single daily dose (3.5 mg/kg/day) for 4 days, Cyclophosphamide 40 mg/kg/day for 3 days. GvHD prophylaxis consisted of Cyclosporin 3 mg/kg/day IV (days -2 to +1), 2 mg/kg/day IV (days +2 to +42), changed to oral administration of 4 mg/kg/day from day +43. Hemodialysis was performed every other day in sterile room. Nucleated marrow cells infused: 4.4 × 108/kg. A mixed chimerism (VNTR more than 90% donor) was documented at day +19. ANC &gt;0.5 × 109/L at day +21. PLT &gt;20 × 109/L at day +15. Eight months after transplantation, patient is in good general condition, with stable mixed chimerism (more than 95% donor), haemoglobin above 11 g/dl, leucocytes and platelets in normal range. Substantial improvement of chronic anaemia ameliorated patient’s quality of life and now patient can be considered a kidney transplant candidate. This single case report does not allow definitive conclusions to be drawn regarding HSCT suitability of patients with chronic renal failure on hemodialysis. We can observe that once-daily intravenous busulfan administration (after the hemodialysis session) has made it possible to keep therapeutic range, as has been ascertained by pharmacokinetic study of busulfan.</jats:p> |
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| author | Argiolu, Francesca, Sanna, Maria Adele, Bartoli, Antonella, Ferrara, Rocco, Piroddi, Antonio, Addari, Carmen, Orofino, Maria Grazia, Rizzo, Fabiana, Cossu, Fausto, Scalas, Maria Rosa, Broglia, Monica, Giagu, Nicolina, Galanello, Renzo |
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| description | <jats:title>Abstract</jats:title> <jats:p>Until now few patients with renal failure on hemodialysis have undergone haematopoietic stem-cell transplantation (HSCT), and none with thalassaemia. Case report. Patient of 45 years, UPN 168, with β Thalassaemia major, genetic compound β0 39 C--&gt;T/β0 6-A, non transfusible due to red blood cell immunization (positive DAT, Hb 5–7 g/dl), under hemodialytic treatment for three years for bilateral focal nephrosclerosis. He suffered from cardiopathy (atrial thrombosis, pulmonary hypertension, atrial and ventricular dilatation), hypothyroidism, hepatopathy (HCV positive with grade II haemosiderosis). Quality of life was much harmed by the severe chronic anaemia and nephropathy. In December 2007 we performed HSCT from his HLA identical brother. Conditioning regimen included: Busulfan (Bu) IV (Busilvex Pierre Fabre Médicament) in single daily dose (3.5 mg/kg/day) for 4 days, Cyclophosphamide 40 mg/kg/day for 3 days. GvHD prophylaxis consisted of Cyclosporin 3 mg/kg/day IV (days -2 to +1), 2 mg/kg/day IV (days +2 to +42), changed to oral administration of 4 mg/kg/day from day +43. Hemodialysis was performed every other day in sterile room. Nucleated marrow cells infused: 4.4 × 108/kg. A mixed chimerism (VNTR more than 90% donor) was documented at day +19. ANC &gt;0.5 × 109/L at day +21. PLT &gt;20 × 109/L at day +15. Eight months after transplantation, patient is in good general condition, with stable mixed chimerism (more than 95% donor), haemoglobin above 11 g/dl, leucocytes and platelets in normal range. Substantial improvement of chronic anaemia ameliorated patient’s quality of life and now patient can be considered a kidney transplant candidate. This single case report does not allow definitive conclusions to be drawn regarding HSCT suitability of patients with chronic renal failure on hemodialysis. We can observe that once-daily intravenous busulfan administration (after the hemodialysis session) has made it possible to keep therapeutic range, as has been ascertained by pharmacokinetic study of busulfan.</jats:p> |
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| spelling | Argiolu, Francesca Sanna, Maria Adele Bartoli, Antonella Ferrara, Rocco Piroddi, Antonio Addari, Carmen Orofino, Maria Grazia Rizzo, Fabiana Cossu, Fausto Scalas, Maria Rosa Broglia, Monica Giagu, Nicolina Galanello, Renzo 0006-4971 1528-0020 American Society of Hematology Cell Biology Hematology Immunology Biochemistry http://dx.doi.org/10.1182/blood.v112.11.4338.4338 <jats:title>Abstract</jats:title> <jats:p>Until now few patients with renal failure on hemodialysis have undergone haematopoietic stem-cell transplantation (HSCT), and none with thalassaemia. Case report. Patient of 45 years, UPN 168, with β Thalassaemia major, genetic compound β0 39 C--&gt;T/β0 6-A, non transfusible due to red blood cell immunization (positive DAT, Hb 5–7 g/dl), under hemodialytic treatment for three years for bilateral focal nephrosclerosis. He suffered from cardiopathy (atrial thrombosis, pulmonary hypertension, atrial and ventricular dilatation), hypothyroidism, hepatopathy (HCV positive with grade II haemosiderosis). Quality of life was much harmed by the severe chronic anaemia and nephropathy. In December 2007 we performed HSCT from his HLA identical brother. Conditioning regimen included: Busulfan (Bu) IV (Busilvex Pierre Fabre Médicament) in single daily dose (3.5 mg/kg/day) for 4 days, Cyclophosphamide 40 mg/kg/day for 3 days. GvHD prophylaxis consisted of Cyclosporin 3 mg/kg/day IV (days -2 to +1), 2 mg/kg/day IV (days +2 to +42), changed to oral administration of 4 mg/kg/day from day +43. Hemodialysis was performed every other day in sterile room. Nucleated marrow cells infused: 4.4 × 108/kg. A mixed chimerism (VNTR more than 90% donor) was documented at day +19. ANC &gt;0.5 × 109/L at day +21. PLT &gt;20 × 109/L at day +15. Eight months after transplantation, patient is in good general condition, with stable mixed chimerism (more than 95% donor), haemoglobin above 11 g/dl, leucocytes and platelets in normal range. Substantial improvement of chronic anaemia ameliorated patient’s quality of life and now patient can be considered a kidney transplant candidate. This single case report does not allow definitive conclusions to be drawn regarding HSCT suitability of patients with chronic renal failure on hemodialysis. We can observe that once-daily intravenous busulfan administration (after the hemodialysis session) has made it possible to keep therapeutic range, as has been ascertained by pharmacokinetic study of busulfan.</jats:p> Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis Blood |
| spellingShingle | Argiolu, Francesca, Sanna, Maria Adele, Bartoli, Antonella, Ferrara, Rocco, Piroddi, Antonio, Addari, Carmen, Orofino, Maria Grazia, Rizzo, Fabiana, Cossu, Fausto, Scalas, Maria Rosa, Broglia, Monica, Giagu, Nicolina, Galanello, Renzo, Blood, Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis, Cell Biology, Hematology, Immunology, Biochemistry |
| title | Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_full | Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_fullStr | Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_full_unstemmed | Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_short | Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| title_sort | haematopoietic stem cell transplantation in thalassaemic patient with renal failure on hemodialysis |
| title_unstemmed | Haematopoietic Stem Cell Transplantation in Thalassaemic Patient with Renal Failure on Hemodialysis |
| topic | Cell Biology, Hematology, Immunology, Biochemistry |
| url | http://dx.doi.org/10.1182/blood.v112.11.4338.4338 |