Journal of Hematology - Supplements - Haematologica

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36 APPENDIX Transplant Centers. Number of unrelated donor transplants by year and principal investigator. No. of BMTs CIC Transplant Centre ≤1994 ≥1995 Total Main investigators • Dipartimento di Ematologia, Ospedale S.Martino - Genova 21 98 119 A. Bacigalupo T. Lamparelli • Clinica Pediatrica, IRCCS Policlinico S. Matteo - Pavia 20 57 77 F. Locatelli G. Giorgiani • U.O. Ematologia-Oncologia Pediatrica, IRCCS G.Gaslini - Genova 14 39 53 G. Dini E. Lanino • Unità TMO, Dipartimento di Ematologia, Ospedale Careggi, Firenze 4 44 48 A. Bosi S. Guidi • Istituto di Ematologia e Oncologia Medica Seragnoli, Ospedale S.Orsola, Università di Bologna 5 39 44 G. Bandini • Dipartimento di Ematologia, Unità TMO, Policlinico S. Matteo, Pavia 8 33 41 E.P. Alessandrino • Clinica Pediatrica, Università di Brescia 10 25 35 F. Porta E. Mazzolari • Clinica Pediatrica, Ospedale S. Gerardo, Monza 4 22 26 C. Uderzo A. Balduzzi • Clinica Pediatrica e Centro Leucemie Infantili, Università di Padova 3 23 26 C. Messina S. Cesaro • Dipartimento di Pediatria, Ospedale Regina Margherita, Univ. di Torino 8 16 24 F. Fagioli E. Vassallo • Dipartimento di Biotecnologie Cellulari e Ematologia, la Sapienza, Roma - 23 23 W. Arcese A. Iori • Dipartimento di Ematologia, Ospedale Niguarda Ca’ Granda, Milano 5 15 20 P. Marenco • Istituto di Ematologia, Università di Udine - 18 18 R. Fanin • Centro TMO, U.O. Ematologia, Azienda Ospedaliera S. Giovanni Torino - 16 16 M. Falda F. Locatelli • Unità TMO, IRCCS Ospedale Maggiore, Milano 4 11 15 D. Soligo E. Tagliaferri • Dipartimento di Scienze Mediche e Genetica Medica, Università di Cagliari 3 11 14 G. La Nasa • Dipartimento di Ematologia e Centro Trapianti, Ospedale di Pesaro 14 14 C. Giardini • Unità TMO, Clinica Pediatrica, Università di Bologna 13 13 A. Pession • Divisione di Ematologia e Unità TMO, Ospedale V. Cervello, Palermo 13 13 R. Scimè • Dipartimento di Ematologia, Ospedale Civile, Pescara 3 9 12 P. Di Bartolomeo • Divisione Ematologia, Ospedale di Bergamo 10 10 T. Barbui A. Rambaldi • Dipartimento di Ematologia, Ospedale S. Bortolo, Vicenza 10 10 F. Rodeghiero • Clinica Pediatrica – Centro Trapianti Ospedale Burlo Garofolo, Trieste 2 4 6 M. Andolina M. Rabusin • Ematologia - Policlinico Borgo Roma, Università di Verona 5 5 F. Benedetti • Centro Trapianti, 2° Clinica Pediatrica, Università di Cagliari 3 3 F. Argiolu • Dipartimento di Ematologia, Policlinico Monteluce, Università di Perugia 3 - 3 F. Aversa • Istituto di Clinica Pediatrica, Università di Pisa 2 2 C. Favre • Azienda Ospedaliera Bianchi-Melacrino- Morelli, Reggio Calabria 2 2 P. Iacopino • Istituto Semeiotica Medica, Ematologia, Università Cattolica Sacro Cuore, Roma 2 2 G. Leone • Divisione di Ematologia, Università di Napoli 1 1 B. Rotoli • Divisione di Ematologia, Ospedale S. Camillo, Roma 1 1 A. De Laurenzi Acknowledgments We acknowledge the skilled care of the physicians, nurses, and transplant coordinators of the bone marrow transplant units who participated in this programme: T. Barbui and A. Rambaldi (Divisione Ematologia, Ospedale di Bergamo), F. Rodeghiero (Divisione di Ematologia, Ospedale di Vicenza), M. Andolina and M. Rabusin (Clinica Pediatrica, Università di Trieste), F. Benedetti (Cattedra di Ematologia, Università di Verona), F. Argiolu (Clinica Pediatrica, Università di Cagliari), F. Aversa (Cattedra di Ematologia, Università di Perugia), C. Favre (Clinica Pediatrica, Università di Pisa), P. Jacopino (Divisione di Ematologia, Ospedale di Reggio Calabria), G. Leone (Divisione di Ematologia, Università Cattolica di Roma), B. Rotoli (Divisione di Ematologia, Ospedale di Napoli), A. De Laurenzi (Divisione di Ematologia, Ospedale S. Camillo di Roma). We thank Valerie Perricone for editorial suggestions and Mirella Berlengiero for the preparation of the manuscript. We thank Rosi Oneto, Roberto Rondelli, Francesca Losito and Barbara Negroni for data managing. This study was supported in part by Associazione Donatori di Midollo Osseo (ADMO) and by Associazione Italiana contro le Leucemie (AIL). haematologica vol. 85(supplement to n. 11):November 2000
haematologica 2000; 85(supplement to n. 11):37-40 original paper Haploidentical bone marrow transplantation in leukemia and genetic diseases M. ANDOLINA, N. MAXIMOVA, M. RABUSIN, D. VUJIC,° G. BUNJEVACKI,° C. VIDALI,* A. BEORCHIA* IRCCS Burlo Garofolo, Trieste; *Unità Operativa di Radioterapia, Trieste, Italy; Mother’s and Child’s Institute, Belgrade, Yugoslavia ABSTRACT From 1986 to June 2000, sixty children suffering from acute and chronic leukemia (n = 42, 33 of which in resistant relapse), genetic diseases (n = 11), aplastic anemia (n = 2, one of which with platelet refractoriness and bleeding), myelodysplasia (n = 5) received an haploidentical bone marrow, mismatched for 2-3 HLA loci. The donor’s marrow was treated in vitro with vincristine and methylprednisolone to obtain a functional T depletion (MLC and CTL inhibition, functional blockade of Th1 and Th2). The prevalence of infectious complications and GVHD was similar to that recorded in matched unrelated donor (MUD) transplants. In situations of high risk of rejection (chronic leukemia, genetic diseases) we infused immediately one half of the harvest and then frozen aliquots from the second week. Of the 25 ALL and 8 AML in resistant relapse, 3 survived, disease-free at 14, 8 and 1 years respectively. Of the 3 ALL, transplanted during remission, 1 is surviving at 18 months. Of the 6 CML, 1 had fractionated bone marrow and is surviving at 3 years, and 5 had standard single dose infusion and died of progression of their disease after rejection of the graft (4) or blast crisis after complete engraftment (1). The 2 patients with aplastic anemia, those with myelodysplasia, and 6 of the 10 with genetic disorders died of transplant-related complications or disease progression. 4 patients with osteopetrosis (n=2), MLD (n=1), Wiskott Aldrich dis. (n=1) survive at 8, 2, 5 and 1.5 years respectively. In patients transplanted with fractionated marrow GVHD >2nd grade occurred in 15%. Only one patient rejected the graft. Compared with MUD transplantation, mismatched BMT whenever performed in patients in good conditions provides similar outcome and widens the donor availability. ©2000, Ferrata Storti Foundation Key words: haploidentical, mismatched bone marrow transplantation, leukemia, genetic diseases About 40 percent of patients fail to find a perfectly matched donor among relatives on in any of the donor registries. 1 Therefore, more than one third of the patients who could be theoretically cured by a bone marrow Correspondence: Andolina Marino, MD, Transplant Department, IRCCS Burlo Garofolo, 34100 Trieste, Italy. Phone: 0039-040-3785276 –Fax: 0039-040-3785494 – Email: andolina@burlo.trieste.it transplantion fails to exploit this treatment. 1,2 It has been shown that transplants using mismatched bone marrow can be as effective as those in which the donor and recipient are fully matched. 7,8 Mismatched bone marrow transplant increases the chances of finding a donor and shortens the time for donor search. In fact, the timing of the transplant is critical especially in patients with ALL in CR2 and genetic diseases for whom the BMT is a urgent procedure. 1,2,11 Haploidentical siblings and parents are suitable donors, whenever healthy and of a proper age and body size. Even if the first experiences of 2-3 loci mismatched transplants were performed more than 15 years ago, 3 this procedure did not obtain a large approval from most centres. However there is an increasing evidence that a mismatched BMT is able to overcome the most severe clinical situations. 4-6 Among strategies of performing the 2-3 loci mismatch transplant and not to perform the transplant when an HLA identical donor is not available, we adopted the former in 1986. Since then, we have performed 60 transplants and the follow up data are similar to those of MUD, with the advantage that any patient is given opportunity for treatment. Selection of the patients In the first series of patients we treated mainly children with resistant relapse of acute leukemia (25). These patients had not only resistant blasts in their marrow and peripheral blood, but in most cases suffered by several untoward effects of chemotherapy (infections, liver damage). Later we began transplanting even chronic leukemias (6), acute leukemias in remission beyond the first (3), genetic diseases (13), severe aplastic anemia (2) and myelodysplastic syndromes(5). Since 1996, the patients who had not previously undergone heavy treatment with immunosuppressive drugs, and therefore were particularly susceptible to rejecting the marrow, received a conditioning regimen (protocol #2) aimed at reducing this danger. haematologica vol. 85(supplement to n. 11):November 2000
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