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Il trapianto di midollo: un passo avanti per le malattie autoimmuni intrattabili
BONE MARROW TRANSPLANTATION IN AUTOIMMUNE DISEAS
M. ANDOLINA, M. RABUSIN, N. MAXIMOVA, S. PARCO, M. CANDUSSO, D. FARAGUNA, L. LEPORE
Clinica Pediatrica, IRCCS “Burlo Garofolo”, Trieste
Aprile 1998 - pagg. 233 -236
Abstract
We report the results of a series of autologous
stem cell transplants in autoimmune
diseases.
The stem cells were treated ex vivo with vincristine
and methylprednisolone as previously
in allogeneic mismatched transplants in
advanced leukemia. The conditioning regimen
was: cyclophosphamide and antilymphocytic
serum (+AraC in multiple
sclerosis). The outcome was very good in 2
over 3 patients with scleroderma secondary
to chronic GVHD (1 relapsed after 6
months), in 3 juvenile chronic arthritis, in 1
myasthenia gravis, 1 multiple sclerosis, 1
pure red cell aplasia (relapsed 5 months later);
1 patient with autoimmune enteropathy
had only laboratory improvements
(absorption, biopsy). 1 patient with systemic
vasculitis and 1 with SLE in CLL seem
improving after a short follow up.
The patients did not suffer from major untoward
effects and were discharged after
10 days in treatment with cyclosporin and
cortison (progressively withdrawn).
Parole chiave
Classificazione MeSH
Bibliografia
stem cell transplants in auto-immune disease:
a consensus report written on behalf of the
European League against Rheumatism (EULAR)
and the European Group for Blood and
Marrow Transplantation (EBMT). Bone Marrow
Transplant Apr, 19, 7, 643-5, 1997.
2. van Bekkum DW: BMT in experimental autoimmune diseases. Bone Marrow Transplant Mar, 11, 3, 183-7, 1993.
3. Tyndall A: Hematopoietic stem cell transplantation in rheumaic diseases other than systemic sclerosis and systemic lupus erythematodes. J Rheum 48 (suppl), 94-97, 1997.
4. Snowden JA, Patton WN, ODonnell JL, Hannah EE, Hart DN: Prolonged remission of longstanding systemic lupus erythematosus after autologous bone marrow transplant for non-Hodgkin’s lymphoma. Bone Marrow Transplant Jun, 19, 12, 1247-50, 1997.
5. Marmont AM, Van Bekkum DW: Stem cell transplantation for severe autoimmune diseases: new proposals but still unanswered questions. Bone Marrow Transplant Oct, 16, 4, 497-8, 1995.
6. Andolina M, Maximova N, Rabusin M, Parco S, Colovic M, Jankovic G: Bone marrow transplantation in chronic GvHD and autoimmune diseases. Bone Marrow Transplant, Nov, 18 (suppl. 2), 145-7, 1996.
8. Marmont AM: Stem cell transplantation for severe autoimmune disorders, with special reference to rheumatic diseases. J Rheumatol Suppl, May, 48, 13-8, 1997.
9. Kapoor N, Keever CA, Leong N, Beck EX: Effects of ex vivo treatment with methylprednisolone and vincristine on bone marrow lymphohematopoietic cells. Prog Clin Biol Res 377, 49-56, 1992.
10. Lanfranchi A, Andolina M, Tettoni K, Porta F, Locatelli F, De Manzini A, Candotti F, Albertini A: Functional depletion of T cells by vincristine and methylprednisolone as an in vitro model for the prevention of graft versus host disease. Haematologica Jan-Feb, 77, 1, 11-5, 1992.
11. Fassas A, Anagnostopulos A, Kazis A, et al: Peripheral blood progenitor cell transplantation for treatment of multiple sclerosis. Bone Marrow Transplant 17 (suppl. 1), 69, 1996.
12. Wulffraat NM, van Royen A, et al: Autologous bone marrow transplantation in refractory polyarticular and systemic JCA. Bone Marrow Transplant 21 (suppl. 1), 179, 1998.
13. Euler HH, Marmont AM, Bacigalupo A, Fastenrath S, Dreger P, Hoffknecht M, Zander AR, Schalke B, Hahn U, Haas R, Schmitz N: Early recurrence or persistence of autoimmune diseases after unmanipulated autologous stem cell transplantation. Blood Nov, 88, 9, 3621-5, 1996.
14. Hahn BH: The potential role of autologous stem cell transplantation in patients with systemic lupus erythematosus. J Rheumatol Suppl May, 48, 89-93, 1997.
15. Joske DJ: Autologous bone marrow transplantation for rheumathoid arthritis. Lancet 337-338, 1997.
16. Snowden JA, Milliken ST, Brooks PM, Biggs JC: High doses cyclophosphamide with autologous stem cell rescue in severe active rheumatoid arthritis. A dose escalation. Bone Marrow Transplant 21 (suppl. 1), 184, 1998.
17. Locatelli F, Ravelli A, Maccario R, et al: Autologous peripheral blood selected CD34+ cell transplantation for the treatment of severe progressive systemic sclerosis. Bone Marrow Transplant 19 (suppl. 1), 208, 1997.
2. van Bekkum DW: BMT in experimental autoimmune diseases. Bone Marrow Transplant Mar, 11, 3, 183-7, 1993.
3. Tyndall A: Hematopoietic stem cell transplantation in rheumaic diseases other than systemic sclerosis and systemic lupus erythematodes. J Rheum 48 (suppl), 94-97, 1997.
4. Snowden JA, Patton WN, ODonnell JL, Hannah EE, Hart DN: Prolonged remission of longstanding systemic lupus erythematosus after autologous bone marrow transplant for non-Hodgkin’s lymphoma. Bone Marrow Transplant Jun, 19, 12, 1247-50, 1997.
5. Marmont AM, Van Bekkum DW: Stem cell transplantation for severe autoimmune diseases: new proposals but still unanswered questions. Bone Marrow Transplant Oct, 16, 4, 497-8, 1995.
6. Andolina M, Maximova N, Rabusin M, Parco S, Colovic M, Jankovic G: Bone marrow transplantation in chronic GvHD and autoimmune diseases. Bone Marrow Transplant, Nov, 18 (suppl. 2), 145-7, 1996.
8. Marmont AM: Stem cell transplantation for severe autoimmune disorders, with special reference to rheumatic diseases. J Rheumatol Suppl, May, 48, 13-8, 1997.
9. Kapoor N, Keever CA, Leong N, Beck EX: Effects of ex vivo treatment with methylprednisolone and vincristine on bone marrow lymphohematopoietic cells. Prog Clin Biol Res 377, 49-56, 1992.
10. Lanfranchi A, Andolina M, Tettoni K, Porta F, Locatelli F, De Manzini A, Candotti F, Albertini A: Functional depletion of T cells by vincristine and methylprednisolone as an in vitro model for the prevention of graft versus host disease. Haematologica Jan-Feb, 77, 1, 11-5, 1992.
11. Fassas A, Anagnostopulos A, Kazis A, et al: Peripheral blood progenitor cell transplantation for treatment of multiple sclerosis. Bone Marrow Transplant 17 (suppl. 1), 69, 1996.
12. Wulffraat NM, van Royen A, et al: Autologous bone marrow transplantation in refractory polyarticular and systemic JCA. Bone Marrow Transplant 21 (suppl. 1), 179, 1998.
13. Euler HH, Marmont AM, Bacigalupo A, Fastenrath S, Dreger P, Hoffknecht M, Zander AR, Schalke B, Hahn U, Haas R, Schmitz N: Early recurrence or persistence of autoimmune diseases after unmanipulated autologous stem cell transplantation. Blood Nov, 88, 9, 3621-5, 1996.
14. Hahn BH: The potential role of autologous stem cell transplantation in patients with systemic lupus erythematosus. J Rheumatol Suppl May, 48, 89-93, 1997.
15. Joske DJ: Autologous bone marrow transplantation for rheumathoid arthritis. Lancet 337-338, 1997.
16. Snowden JA, Milliken ST, Brooks PM, Biggs JC: High doses cyclophosphamide with autologous stem cell rescue in severe active rheumatoid arthritis. A dose escalation. Bone Marrow Transplant 21 (suppl. 1), 184, 1998.
17. Locatelli F, Ravelli A, Maccario R, et al: Autologous peripheral blood selected CD34+ cell transplantation for the treatment of severe progressive systemic sclerosis. Bone Marrow Transplant 19 (suppl. 1), 208, 1997.