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101. A phase III, open-label, randomized comparison of AS1413 (amonafide L-malate) plus cytarabine with daunorubicin plus cytarabine in secondary acute myeloid leukemia (ACCEDE).

Author

Stone, R. M., primary, Allen, S. L., additional, Pigneux, A., additional, Stuart, R. K., additional, Wetzler, M., additional, Rizzieri, D., additional, Erba, H. P., additional, Damon, L. E., additional, Jang, J. H., additional, Tallman, M. S., additional, Warzocha, K., additional, Masszi, T., additional, Sekeres, M. A., additional, Miklos, E., additional, Horst, H., additional, Selleslag, D. L. D., additional, Solomon, S. R., additional, Venugopal, P., additional, Lundberg, A. S., additional, and Powell, B. L., additional

Published
2011
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103. A randomized, multicenter, phase (Ph) III study comparing carfilzomib (CFZ), lenalidomide (LEN), and dexamethasone (Dex) to LEN and Dex in patients (Pts) with relapsed multiple myeloma (MM).

Author

Moreau, P., primary, Palumbo, A. P., additional, Stewart, A. K., additional, Rajkumar, V., additional, Jakubowiak, A. J., additional, Halka, K., additional, Goranov, S., additional, Bumbea, H., additional, Pendergrass, K. B., additional, Lupu, A., additional, Dimopoulos, A., additional, Rocafiguera, A. O., additional, Gandhi, J. G., additional, Mihaylov, G., additional, Masszi, T., additional, Matous, J., additional, Fonseca, G., additional, Bryce, R., additional, and Siegel, D. S. D., additional

Published
2011
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104. Plerixafor for PBSC mobilisation in myeloma patients with advanced renal failure: safety and efficacy data in a series of 21 patients from Europe and the USA

Author

Douglas, K W, primary, Parker, A N, additional, Hayden, P J, additional, Rahemtulla, A, additional, D'Addio, A, additional, Lemoli, R M, additional, Rao, K, additional, Maris, M, additional, Pagliuca, A, additional, Uberti, J, additional, Scheid, C, additional, Noppeney, R, additional, Cook, G, additional, Bokhari, S W, additional, Worel, N, additional, Mikala, G, additional, Masszi, T, additional, Taylor, R, additional, and Treisman, J, additional

Published
2011
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105. Safety of omacetaxine mepesuccinate (OM) subcutaneous (SQ) injection for the treatment of chronic myeloid leukemia (CML) patients (pts) resistant or intolerant to tyrosine kinase inhibitors (TKIs): Analysis of two phase II studies.

Author

Lipton, J. H., primary, Wetzler, M., additional, Nicolini, F., additional, Baccarani, M., additional, Baer, M. R., additional, Masszi, T., additional, Cram, D., additional, Benichou, A., additional, Nanda, N., additional, and Cortes, J. E., additional

Published
2010
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106. A166 VMP in Renally Impaired Newly Diagnosed MM Patients: Cohort Analysis of the VISTA Study

Author

Dimopoulos, MA, primary, Richardson, PG, additional, Schlag, R, additional, Khuageva, NK, additional, Shpilberg, O, additional, Kastritis, E, additional, Kropff, M, additional, Petruci, M, additional, Delforge, M, additional, Alexeeva, J, additional, Schots, R, additional, Masszi, T, additional, Mateos, MV, additional, Deraedt, V, additional, Liu, K, additional, Cakana, A, additional, van de Velde, H, additional, and San Miguel, J, additional

Published
2009
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109. P077 NPM1 and FLT3 mutations in acute myeloid leukemia

Author

Nahajevszky, S., primary, Tordai, A., additional, Szilvasi, A., additional, Meggyesi, N., additional, Adam, E., additional, Kozma, A., additional, Lueff, S., additional, Lovas, N., additional, Kapas, B., additional, Matrai, Z., additional, Sipos, A., additional, Masszi, T., additional, and Andrikovics, H., additional

Published
2007
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111. O37 BCR-ABL kinase domain mutations and clonal evolution as major causes of resistance to targeted therapy in chronic myelogenous leukemia

Author

Andrikovics, H., primary, Meggyesi, N., additional, Lueff, S., additional, Batai, A., additional, Adam, E., additional, Kozma, A., additional, Halm, G., additional, Nahajevszky, S., additional, Kapás, B., additional, Csukly, Z., additional, Lovas, N., additional, Reményi, P., additional, Tordai, A., additional, and Masszi, T., additional

Published
2007
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116. O21 A retrospective single-center experience with bortezomib-based therapy of relapsed and/or refractory multiple myeloma: survival results of our first 104 consecutive patients

Author

Mikala, G., primary, Batai, A., additional, Bodo, I., additional, Csukly, Z., additional, Dolgos, J., additional, Halm, G., additional, Janosi, J., additional, Kapas, B., additional, Lovas, N., additional, Lueff, S., additional, Peto, M., additional, Remenyi, P., additional, Sipos, A., additional, Toth, Z., additional, and Masszi, T., additional

Published
2007
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119. P061 Type 3 von Willebrand disease in Hungary: A partial large deletion is the most common genetic defect

Author

Mohl, A., primary, Boda, Z., additional, Jager, R., additional, Losonczy, H., additional, Marosi, A., additional, Masszi, T., additional, Nagy, E., additional, Nemes, L., additional, Obser, T., additional, Oyen, F., additional, Radványi, G., additional, Sallai, K., additional, Schlammadinger, Á., additional, Szélessy, Zs., additional, Vezendy, K., additional, Schneppenheim, R., additional, and Bodó, I., additional

Published
2007
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120. HIGH NOREPINEPHRINE LEVEL IS ASSOCIATED WITH SIGNIFICANT PLATELET HYPERREACTIVITY DEMONSTRATED BY INCREASED PLATELET P-SELECTIN POSITIVITY, ADENOSIN DIPHOSPHATE AND COLLAGEN INDUCED AGGREGATIONS IN STABLE CORONARY HEART DISEASE PATIENTS ON DUAL ANTIPLATEL

Author

Béres, B.J., primary, Tóth-Zsámboki, E., additional, Vargová, K., additional, Kiss, N., additional, Kovács, A., additional, Husvéth, Á., additional, László, Á., additional, Masszi, T., additional, Préda, I., additional, and Kiss, R.G., additional

Published
2007
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121. Syngeneic transplantation in multiple myeloma - a case-matched comparison with autologous and allogeneic transplantation. European Group for Blood and Marrow Transplantation.

Author

Gahrton, G, Svensson, H, Bjorkstrand, B, Apperley, C, Carlson, K, Cavo, M, Ferrant, A, Fouillard, L, Gratecos, N, Gratwohl, A, Guilhot, F, Lambertenghi Deliliers, G, Ljungman, P, Masszi, T, Milligan, DW, Powles, RL, Reiffers, J, Samson, JD, Stoppa, AM, Vernant, JP, Volin, L, Wallvik, J, Gahrton, G, Svensson, H, Bjorkstrand, B, Apperley, C, Carlson, K, Cavo, M, Ferrant, A, Fouillard, L, Gratecos, N, Gratwohl, A, Guilhot, F, Lambertenghi Deliliers, G, Ljungman, P, Masszi, T, Milligan, DW, Powles, RL, Reiffers, J, Samson, JD, Stoppa, AM, Vernant, JP, Volin, L, and Wallvik, J

Published
1999

122. European results of unrelated cord blood transplants. Eurocord group

Author

Gluckman, E., Rocha, V., Chastang, C., Abecasis, M., Arcese, W., Badell Serra, I., Beksac, M., Bernaudin, F., Bogdanic, V., Bosi, A., Cahn, J. Y., Cornu, G., Delliers, L. G., Dokal, I., Favre, C., Fernandez, M. N., Ferreira, E., Fisher, A., Gibson, B. E. S., Jedrzejcack, W., Jouet, J. P., Kinoshita, A., Kobylka, P., Laporte, J. P., Locatelli, Franco, Lutz, P., Masszi, T., Millone, J., Milovic, V., Miniero, R., Nagler, A., Ortega, J. J., Pasquini, R., Pension, A., Perez-Oyteza, J., Peters, C., Plouvier, E., Reiffers, J., Roberts, I., Roittman, S., Saarinen, U., Souillet, G., Stary, J., Takaue, Y., Urban, C., Veys, P., Vilmer, E., Vossen, J. M., Yaniv, I., Zintl, F., Locatelli F. (ORCID:0000-0002-7976-3654), Gluckman, E., Rocha, V., Chastang, C., Abecasis, M., Arcese, W., Badell Serra, I., Beksac, M., Bernaudin, F., Bogdanic, V., Bosi, A., Cahn, J. Y., Cornu, G., Delliers, L. G., Dokal, I., Favre, C., Fernandez, M. N., Ferreira, E., Fisher, A., Gibson, B. E. S., Jedrzejcack, W., Jouet, J. P., Kinoshita, A., Kobylka, P., Laporte, J. P., Locatelli, Franco, Lutz, P., Masszi, T., Millone, J., Milovic, V., Miniero, R., Nagler, A., Ortega, J. J., Pasquini, R., Pension, A., Perez-Oyteza, J., Peters, C., Plouvier, E., Reiffers, J., Roberts, I., Roittman, S., Saarinen, U., Souillet, G., Stary, J., Takaue, Y., Urban, C., Veys, P., Vilmer, E., Vossen, J. M., Yaniv, I., Zintl, F., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

This report summarizes the results of unrelated cord blood transplants. Sixty-five patients were reported to Eurocord. One year survival was 29%. Factors associated with survival were diagnosis, inborn errors had a better survival than leukemia or aplastic anemia; higher number of cells infused and negative pre-transplant CMV serology were also favorable factors. Granulocyte and platelet engraftment were associated with a higher number of nucleated cells infused, GVH incidence was low especially in CMV negative patients, it was not associated with the degree of HLA disparity. These results were similar to other series reported in the USA and showed the benefit of using mismatched unrelated cord blood in clearly defined situations.

Published
1998

127. Unrelated BMT in Hungary, 4 years experience

Author

Gyódi, É., primary, Nyán, E., additional, Kotlán, B., additional, Rajczy, K., additional, Pénzes, M., additional, Dénes, R., additional, Masszi, T., additional, Tímár, L., additional, and Petrányi, Gy., additional

Published
1996
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128. Superiority of the Triple Combination of Bortezomib-Thalidomide-Dexamethasone Over the Dual Combination of Thalidomide-Dexamethasone in Patients With Multiple Myeloma Progressing or Relapsing After Autologous Transplantation: The MMVAR/IFM 2005-04...

Author

Garderet L, Iacobelli S, Moreau P, Dib M, Lafon I, Niederwieser D, Masszi T, Fontan J, Michallet M, Gratwohl A, Milone G, Doyen C, Pegourie B, Hajek R, Casassus P, Kolb B, Chaleteix C, Hertenstein B, Onida F, and Ludwig H

Published
2012
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129. Dasatinib in imatinib-resistant or imatinib-intolerant chronic myeloid leukemia in blast phase after 2 years of follow-up in a phase 3 study: efficacy and tolerability of 140 milligrams once daily and 70 milligrams twice daily.

Author

Saglio G, Hochhaus A, Goh YT, Masszi T, Pasquini R, Maloisel F, Erben P, Cortes J, Paquette R, Bradley-Garelik MB, Zhu C, Dombret H, Saglio, Giuseppe, Hochhaus, Andreas, Goh, Yeow Tee, Masszi, Tamas, Pasquini, Ricardo, Maloisel, Frederic, Erben, Philipp, and Cortes, Jorge

Abstract

Background: In a phase 3 study, the authors assessed the effects of dasatinib at doses of 140 mg once daily and 70 mg twice daily in patients who had either chronic myeloid leukemia (CML) in advanced phases or Philadelphia chromosome-positive acute lymphoblastic leukemia and were resistant or intolerant to imatinib. In the current report, the results for patients with CML in blast phase after 2 years of follow-up are reported.Methods: Patients were stratified according to whether they had CML in myeloid blast phase (MBP-CML) or in lymphoid blast phase (LBP-CML) and were randomized (1:1) within each stratum to receive either oral dasatinib 140 mg once daily or 70 mg twice daily.Results: In patients with MBP-CML, the major hematologic response rate was 28% for both regimens; and, in patients with LBP-CML, the major hematologic response rate was 42% for once-daily dasatinib and 32% for twice-daily dasatinib. The major cytogenetic response rates were 25% for once-daily dasatinib and 28% for twice-daily dasatinib in patients with MBP-CML, and the respective rates in patients with LBP-CML were 50% and 40%. The overall survival rate at 24 months was 24% for once-daily dasatinib and 28% for twice-daily dasatinib in patients with MBP-CML, and the respective values in patients with LBP-CML were 21% and 16%. Adverse events indicated a trend toward improved tolerability for the once-daily regimen.Conclusions: The current results suggested that dasatinib 140 mg once daily had similar efficacy and improved tolerability relative to the 70-mg twice-daily regimen in patients with imatinib-resistant, blast phase CML. [ABSTRACT FROM AUTHOR]

Published
2010
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130. Common large partial VWFgene deletion does not cause alloantibody formation in the Hungarian type 3 von Willebrand disease population

Author

MOHL, A., BODA, Z., JAGER, R., LOSONCZY, H., MAROSI, A., MASSZI, T., NAGY, E., NEMES, L., OBSER, T., OYEN, F., RADVÁNYI, G., SCHLAMMADINGER, Á., SZÉLESSY, ZS., VÁRKONYI, A., VEZENDY, K., VILIMI, B., SCHNEPPENHEIM, R., and BODÓ, I.

Abstract

Background:Type 3 von Willebrand disease (VWD) is an autosomal recessive bleeding disorder, characterized by virtually undetectable plasma von Willebrand factor (VWF) and consequently reduced plasma factor VIII levels. Genetic mutations responsible for type 3 VWD are very heterogeneous, scattered throughout the VWFgene and show high variability among different populations. Methods:Twenty‐five severe VWD patients were studied by direct sequencing of the 51 coding exons of the VWFgene. The total number of VWD type 3 families in Hungary is 24, of which 23 were investigated. Results:Fifteen novel mutations were identified in 31 alleles, five being nonsense mutations (p.Q1238X, p.Q1898X, p.Q1931X, p.S2505X and p.S2568X), four small deletions and insertions resulting in frame shifts (c.1992insC, c.3622delT, c.5315insGA and c.7333delG), one a large partial deletion (delExon1‐3) of the 5′‐region, four candidate missense mutations (p.C35R, p.R81G, p.C295S, p.C623T) and one a candidate splice site mutation (c.1730–10C>A). Six previously described mutations were detected in 17 alleles, including the repeatedly found c.2435delC, p.R1659X and p.R1853X. Only one patient developed alloantibodies to VWF, carrying a homozygous c.3622delT. Conclusion:We report the genetic background of the entire Hungarian type 3 VWD population. A large novel deletion, most probably due to a founder effect, seems to be unique to Hungarian type 3 VWD patients with high allele frequency. In contrast to previous reports, none of the five patients homozygous for the large partial deletion developed inhibitors to VWF. This discrepancy raises the possibility of selection bias in some of the reports.

Published
2011
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131. Serum alpha 2-HS glycoprotein concentration in patients with hematological malignancies. A follow-up study.

Author

Kalabay, L, Cseh, K, Benedek, S, Fekete, S, Masszi, T, Herjeczki, K, Pozsonyi, T, and Jakab, L

Subjects
ANTHROPOMETRY, BLOOD proteins, COMPARATIVE studies, GENITOURINARY diseases, GLYCOPROTEINS, INFECTION, LEUKEMIA, LIVER, LONGITUDINAL method, LUNG diseases, LYMPHOMAS, RESEARCH methodology, MEDICAL cooperation, DISEASES in men, MULTIPLE myeloma, MYELOFIBROSIS, RESEARCH, SPLEEN, EVALUATION research, DISEASE complications
Abstract

We observed significantly reduced serum alpha 2-HS glycoprotein concentrations in patients with acute lymphocytic, acute nonlymphocytic, chronic granulocytic and chronic myelomonocytic leukemias, Hodgkin's and non-Hodgkin's lymphomas, myelofibrosis, and multiple myeloma, but not in patients with chronic lymphocytic leukemia and polycythemia vera, as compared with healthy controls. We followed the serum level of the protein for 18 months. Patients with infectious complications, those receiving cytostatic treatment, and those in the preterminal period had further reduced serum alpha 2-HS glycoprotein levels. The reduction of serum alpha 2-HS glycoprotein concentration was primarily due to decreased production caused by infiltration of the liver, a hepatotoxic effect of cytostatic treatment, and, to a lesser degree, to increased consumption. We found statistically significant negative correlations between serum alpha 2-HS glycoprotein concentration and erythrocyte sedimentation rate, serum aspartate aminotransferase and alkaline phosphatase activities, and IgG and IgM concentrations. The determination of the alpha 2-HS glycoprotein concentration is useful for the assessment and follow-up of the clinical status and therapy of patients with hematological malignancies and also has prognostic significance. [ABSTRACT FROM AUTHOR]

Published
1991

132. Serum α2-HS glycoprotein concentration in patients with hematological malignancies

Author

Kalabay, L., Cseh, K., Benedek, S., Fekete, S., Masszi, T., Herjeczki, K., Pozsonyi, T., Jakab, L., and Jakab, L.

Abstract

Summary We observed significantly reduced serum a2-HS glycoprotein concentrations in patients with acute lymphocytic, acute nonlymphocytic, chronic granulocytic and chronic myelomonocytic leukemias, Hodgkin's and non-Hodgkin's lymphomas, myelofibrosis, and multiple myeloma, but not in patients with chronic lymphocytic leukemia and polycythemia vera, as compared with healthy controls. We followed the serum level of the protein for 18 months. Patients with infectious complications, those receiving cytostatic treatment, and those in the preterminal period had further reduced serum a2-HS glycoprotein levels. The reduction of serum a2-HS glycoprotein concentration was primarily due to decreased production caused by infiltration of the liver, a hepatotoxic effect of cytostatic treatment, and, to a lesser degree, to increased consumption. We found statistically significant negative correlations between serum a2-HS glycoprotein concentration and erythrocyte sedimentation rate, serum aspartate aminotransferase and alkaline phosphatase activities, and IgG and IgM concentrations. The determination of the a2-HS glycoprotein concentration is useful for the assessment and follow-up of the clinical status and therapy of patients with hematological malignancies and also has prognostic significance.

Published
1991
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133. Frontline nilotinib in patients with chronic myeloid leukemia in chronic phase: results from the European ENEST1st study

Author

Hochhaus, A., Rosti, G., Cross, N. C. P., Steegmann, J. L., Le Coutre, Philipp, Ossenkoppele, G., Petrov, L., Masszi, T., Hellmann, A., Griskevicius, L., Wiktor-Jedrzejczak, W., Rea, D., Coriu, D., Brümmendorf, Tim Henrik, Porkka, K., Saglio, G., Gastl, G., Müller, M. C., Schuld, P., Di Matteo, P., Pellegrino, A., Dezzani, L., Mahon, F.-X., Baccarani, M., and Giles, F. J.

Subjects
3. Good health
Abstract

Leukemia : normal and malignant hemopoiesis 30(1), 57-64 (2016). doi:10.1038/leu.2015.270, Published by Nature Publ. Group, Basingstoke

135. CARFILZOMIB AND DEXAMETHASONE VS SUBCUTANEOUS BORTEZOMIB AND DEXAMETHASONE IN PATIENTS WITH RELAPSED OR REFRACTORY MULTIPLE MYELOMA: SECONDARY ANALYSIS FROM THE PHASE 3 STUDY ENDEAVOR (NCT01568866)

Author

Goldschmidt, H., Moreau, P., Palumbo, A., Joshua, U., Pour, L., Roman Hajek, Facon, T., Ludwig, H., Niesvizky, R., Oriol, A., Rosinol, L., Straub, J., Suvorov, A., Araujo, C., Rimashevskaya, E., Pika, T., Gaidano, G., Weisel, K., Goronova-Marinova, V., Schwarer, A., Minuk, L., Masszi, T., Karamanesht, I., Offidani, M., Hungria, V., Spencer, A., Orlowski, R., Iskander, K., Feng, S., Aggarwal, S., Chng, W. J., and Dimopoulos, Ma

136. EFFICACY AND SAFETY BY CYTOGENETIC RISK STATUS: PHASE 3 STUDY (ASPIRE) OF CARFILZOMIB, LENALIDOMIDE AND DEXAMETHASONE VERSUS LENALIDOMIDE AND DEXAMETHASONE IN PATIENTS WITH RELAPSED MULTIPLE MYELOMA

Author

Avet-Loiseaul, H., Fonseca, R., Siegei, D., Dimopoulos, M., Spicka, I., Masszi, T., Hajek, R., Rosinol, L., Garanava-Marinova, V., Mihaylavlo, G., Vladimir Maisnar, Mateos, M. V., Wang, M., Niesvizky, R., Oriol, A., Jakubowiak, A., Minarik, J., Palumbo, A., Bensinger, W., Kukreti, V., Ben-Yehuda, D., Tonda, M., Obreja, M., and Moreau, P.

137. EFFECT OF CARFILZOMIB, LENALIDOMIDE, AND DEXAMETHASONE VS LENALIDOMIDE AND DEXAMETHASONE IN PATIENTS WITH RELAPSED MULTIPLE MYELOMA BY LINE OF THERAPY: INTERIM RESULTS FROM THE PHASE 3 ASPIRE STUDY

Author

Dimopoulos, M. A., Stewart, A. K., Rajkumar, S. V., Masszi, T., Spicka, I., Oriol, A., Roman Hajek, Rosinol, L., Siegel, D., Mihaylov, G. G., Goranova-Marinova, V., Rajnics, P., Suvorov, A., Niesvizky, R., Jakubowiak, A., San-Miguel, J., Ludwig, H., Zojwalla, N., Tonda, M., Yang, X., Moreau, P., and Palumbo, A.

139. CARFILZOMIB AND DEXAMETHASONE IMPROVES PROGRESSION-FREE SURVIVAL AND RESPONSE RATES VS BORTEZOMIB AND DEXAMETHASONE IN PATIENTS (PTS) WITH RELAPSED MULTIPLE MYELOMA (RMM): THE PHASE 3 STUDY ENDEAVOR

Author

Dimopoulos, M., Moreau, P., Palumbo, A., Joshua, D., Pour, L., Roman Hajek, Facon, T., Ludwig, H., Oriol, A., Goldschmidt, H., Rosinol, L., Straub, J., Suvorov, A., Araujo, C., Rimashevskaya, E., Pika, T., Gaidano, G., Weisel, K., Goranova-Marinova, V., Schwarer, A., Minuk, L., Masszi, T., Karamanesht, I., Offidani, M., Hungria, V., Spencer, A., Gillenwater, H., Mohamed, N., Feng, S., and Chng, W. J.

140. CARFILZOMIB, LENALIDOMIDE AND DEXAMETHASONE VS LENALIDOMIDE AND DEXAMETHASONE IN MULTIPLE MYELOMA IN RELAPSE ACCORDING TO TREATMENT LINE: INTERMEDIATE SECONDARY ANALYSIS OF ASPIRE (PHASE 3, NCT01080391)

Author

Oriol, A., Rosinol, L., San-Miguel, J., Meletios, Dimopoulos A., Stewart, A. K., Rajkumar, S., Masszi, T., Roman Hajek, Siegel, D., Georgi, Mihaylov G., Goranova-Marinova, V., Rajnics, P., Suvorov, A., Niesvizky, R., Jakubowiak, A., Ludwig, H., Zojwalla, N., Moreau, P., and Palumbo, A.

141. European results of unrelated cord blood transplants

Author

Gluckman, E., Rocha, V., Chastang, C., Abecasis, M., Arcese, W., Isabel Badell, Beksac, M., Bernaudin, F., Bogdanic, V., Bosi, A., Cahn, J. Y., Cornu, G., Delliers, L. G., Dokal, I., Favre, C., Fernandez, M. N., Ferreira, E., Fisher, A., Gibson, B. E. S., Jedrzejcack, W., Jouet, J. P., Kinoshita, A., Kobylka, P., Laporte, J. P., Locatelli, F., Lutz, P., Masszi, T., Millone, J., Milovic, V., Miniero, R., Nagler, A., Ortega, J. J., Pasquini, R., Pension, A., Perez-Oyteza, J., Peters, C., Plouvier, E., Reiffers, J., Roberts, I., Roittman, S., Saarinen, U., Souillet, G., Stary, J., Takaue, Y., Urban, C., Veys, P., Vilmer, E., Vossen, J. M., Yaniv, I., and Zintl, F.

Subjects
Cord blood transplant

142. Allogeneic bone marrow transplantation for acute leukemia in adults

Author

Masszi T, Péter Reményi, Kriván G, Goda V, and Réti M

Subjects
Adult, Male, Leukemia, Myeloid, Acute, Transplantation Conditioning, Treatment Outcome, Adolescent, Humans, Female, Middle Aged, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Bone Marrow Transplantation
Abstract

From September 1993 to August 1997, 24 consecutive adult acute leukemia patients (20 AML and 4 ALL) received allogeneic bone marrow transplant (21 sibling, 1 twin, 2 MUD). The probability of 3 year leukemia free survival is 19/24 (79%), the transplant related mortality is 2/24 (8%), the relapse rate is 3/24 (13%). The median follow up period is 34 months (range 7-51). Three AML patients with high probability of TRM received a special radiation-free conditioning regimen (mitobronitol/cytarabine/ cyclophosphamide) originally described by Kelemen et al in CML patients for decreasing transplant related complications. All the three patients are alive and disease free over 3 years.

144. CLINICAL USEFULNESS OF TC-99M-SESTAMIBI SCINTIGRAPHY IN MULTIPLE MYELOMA.

Author

Varga, Z., Nagy, Z., Buga, K., Moravszki, M., Mikala, G., Masszi, T., and Szilvási, I.

Abstract

Background: Tc-99m-sestamibi (MIBI) scintigraphy (SG) is used to study various malignancies. Usefulness in the work-up of patients with multiple myeloma (MM) has been recently reported. Aim: was to asses clinical value of MIBI SG in different clinical stages of MM. Material and methods: 70 patients with MM had whole body MIBI SG (10 minutes after i.v. injection of 740 MBq MIBI) in the last four years, 14 of them (6 M, 8 F, median age of 59.8 years) had multiple scans in their follow-up. 3 of the 14 patients had monoclonal gammopathy of uncertain significance (MGUS), 3 patients were in complete remission (CR), 6 were in partial remission (PR), 6 had incipient relapse (IR) and 9 had clinically active stage of MM (CA). Clinical activity was determined by serum LDH, electrophoresis, and plasma cell content of the bone marrow. Results: All 9 scans of the 3 patients with MGUS were normal. Normal MIBI distribution was found also in the 3 patients with CR. All patients with PR (6 cases) or IR (6 cases) had circumscribed lesions (2 of them were extramedullary) and 7 of them had moderately increased diffuse activity in the bone marrow. In all 9 CA cases intensive diffuse MIBI activity in the bone marrow was found and 8 patients had circumscribed lesions too. Conclusions: Whole body MIBI-SG is useful to assess activity of MM in clinically equivocal cases, and it may detect extramedullary lesions as well. [ABSTRACT FROM AUTHOR]

Published
2007

148. Effectiveness of intra-arterial steroid administration for the treatment of steroid-refractory acute gastrointestinal graft-versus-host disease.

Author

Bérczi, V., Tóth, A., Tatai, G., Fábián, J., Reményi, P., and Masszi, T.

Subjects
*GRAFT versus host disease, *INTRA-arterial injections, *STEROID drugs, *DRUG administration, *GASTROINTESTINAL diseases, *RETROSPECTIVE studies
Abstract

Aim: To retrospectively assess the clinical effectiveness of intra-arterial steroid administration (IASA) treatment in adult patients who developed steroid-refractory gastrointestinal acute graft-versus-host disease (GI-aGvHD) (≥stage II) following haematopoietic stem cell transplantation.Materials and Methods: Clinical data of 10 consecutive adult patients (age range, 19-61 years; mean age, 42 years) of a single centre with GI-aGvHD (≥stage II) who showed no response to intravenous methylprednisolone and received IASA into the superior (SMA) and/or inferior mesenteric arteries (IMA) were analysed. The severity of aGvHD was determined as the volume of diarrhoea (stages 0-IV) and the Glucksberg grading system before and 12±3 SD, 27±4 and 54±6 days after IASA treatment. Median follow-up was 65 days (range, 22-370 days).Results: Six out of 10 patients at 12 days, 8/10 patients at 27 days, 6/10 patients at 54 days after IASA showed gastrointestinal response. Among them, 1/10 patients at 12 days, 4/10 patients at 27 days, and the same 4/10 patients at 54 days showed complete resolution of GI-aGvHD. The 4/10 patients who reached complete resolution of GI-aGvHD at day 12 or 27 showed a sustained symptom-free state. One in 10 patient showed only a temporary response, 5/10 patients died between days 22 and 67.Conclusions: IASA seems to be a potentially useful second-line therapy for intravenous steroid-refractory GI-aGvHD. [ABSTRACT FROM AUTHOR]

Published
2019
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149. Long-term efficacy and safety of ruxolitinib versus best available therapy in polycythaemia vera (RESPONSE): 5-year follow up of a phase 3 study

Author

Elisa Rumi, Tamás Masszi, Ruben A. Mesa, Jean-Jacques Kiladjian, Francesco Passamonti, Tuochuan Dong, Nathalie Francillard, Vittorio Rosti, Srdan Verstovsek, Beatriz Moiraghi, Carlos Besses, Simon Durrant, Masayuki Hino, Pierre Zachee, Fabrizio Pane, Claire N. Harrison, Martin Griesshammer, Alessandro M. Vannucchi, Monika Wroclawska, Keita Kirito, Carole B. Miller, Igor Wolfgang Blau, Kiladjian, J. -J., Zachee, P., Hino, M., Pane, F., Masszi, T., Harrison, C. N., Mesa, R., Miller, C. B., Passamonti, F., Durrant, S., Griesshammer, M., Kirito, K., Besses, C., Moiraghi, B., Rumi, E., Rosti, V., Blau, I. W., Francillard, N., Dong, T., Wroclawska, M., Vannucchi, A. M., and Verstovsek, S.

Subjects
Ruxolitinib, Time Factors, Polyethylene Glycol, Polyethylene Glycols, 0302 clinical medicine, hemic and lymphatic diseases, Antineoplastic Combined Chemotherapy Protocols, Hydroxyurea, Medicine, Polycythemia Vera, education.field_of_study, Fibrinolytic Agent, Pipobroman, Hematology, Recombinant Protein, Prognosis, Recombinant Proteins, Survival Rate, 030220 oncology & carcinogenesis, Drug Therapy, Combination, Human, medicine.drug, medicine.medical_specialty, Polycythaemia, Prognosi, Population, Interferon alpha-2, Antiviral Agents, Article, Follow-Up Studie, 03 medical and health sciences, Pharmacotherapy, Fibrinolytic Agents, Internal medicine, Nitriles, Humans, Adverse effect, education, Survival rate, Antiviral Agent, Antineoplastic Combined Chemotherapy Protocol, business.industry, Quinazoline, Interferon-alpha, Anagrelide, medicine.disease, Pyrimidines, Pyrazole, Quinazolines, Pyrazoles, business, Follow-Up Studies, 030215 immunology
Abstract

Summary Background Polycythaemia vera is a myeloproliferative neoplasm characterised by excessive proliferation of erythroid, myeloid, and megakaryocytic components in the bone marrow due to mutations in the Janus kinase 2 (JAK2) gene. Ruxolitinib, a JAK 1 and JAK 2 inhibitor, showed superiority over best available therapy in a phase 2 study in patients with polycythaemia vera who were resistant to or intolerant of hydroxyurea. We aimed to compare the long-term safety and efficacy of ruxolitinib with best available therapy in patients with polycythaemia vera who were resistant to or intolerant of hydroxyurea. Methods We report the 5-year results for a randomised, open-label, phase 3 study (RESPONSE) that enrolled patients at 109 sites across North America, South America, Europe, and the Asia-Pacific region. Patients (18 years or older) with polycythaemia vera who were resistant to or intolerant of hydroxyurea were randomly assigned 1:1 to receive either ruxolitinib or best available therapy. Patients randomly assigned to the ruxolitinib group received the drug orally at a starting dose of 10 mg twice a day. Single-agent best available therapy comprised hydroxyurea, interferon or pegylated interferon, pipobroman, anagrelide, approved immunomodulators, or observation without pharmacological treatment. The primary endpoint, composite response (patients who achieved both haematocrit control without phlebotomy and 35% or more reduction from baseline in spleen volume) at 32 weeeks was previously reported. Patients receiving best available therapy could cross over to ruxolitinib after week 32. We assessed the durability of primary composite response, complete haematological remission, overall clinicohaematological response, overall survival, patient-reported outcomes, and safety after 5-years of follow-up. This study is registered with ClinicalTrials.gov , NCT01243944 . Findings We enrolled patients between Oct 27, 2010, and Feb 13, 2013, and the study concluded on Feb 9, 2018. Of 342 individuals screened for eligibility, 222 patients were randomly assigned to receive ruxolitinib (n=110, 50%) or best available therapy (n=112, 50%). The median time since polycythaemia vera diagnosis was 8·2 years (IQR 3·9–12·3) in the ruxolitinib group and 9·3 years (4·9–13·8) in the best available therapy group. 98 (88%) of 112 patients initially randomly assigned to best available therapy crossed over to receive ruxolitinib and no patient remained on best available therapy after 80 weeks of study. Among 25 primary responders in the ruxolitinib group, six had progressed at the time of final analysis. At 5 years, the probability of maintaining primary composite response was 74% (95% CI 51–88). The probability of maintaining complete haematological remission was 55% (95% CI 32–73) and the probability of maintaining overall clinicohaematological responses was 67% (54–77). In the intention-to-treat analysis not accounting for crossover, the probability of survival at 5 years was 91·9% (84·4–95·9) with ruxolitinib therapy and 91·0% (82·8–95·4) with best available therapy. Anaemia was the most common adverse event in patients receiving ruxolitinib (rates per 100 patient-years of exposure were 8·9 for ruxolitinib and 8·8 for the crossover population), though most anaemia events were mild to moderate in severity (grade 1 or 2 anaemia rates per 100 patient-years of exposure were 8·0 for ruxolitinib and 8·2 for the crossover population). Non-haematological adverse events were generally lower with long-term ruxolitinib treatment than with best available therapy. Thromboembolic events were lower in the ruxolitinib group than the best available therapy group. There were two on-treatment deaths in the ruxolitinib group. One of these deaths was due to gastric adenocarcinoma, which was assessed by the investigator as related to ruxolitinib treatment. Interpretation We showed that ruxolitinib is a safe and effective long-term treatment option for patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea. Taken together, ruxolitinib treatment offers the first widely approved therapeutic alternative for this post-hydroxyurea patient population. Funding Novartis Pharmaceuticals Corporation.

Published
2020

150. The potential role of HLA-DRB1*11 in the development and outcome of haematopoietic stem cell transplantation-associated thrombotic microangiopathy.

Author

Balassa, K, Andrikovics, H, Remenyi, P, Batai, A, Bors, A, Kiss, K P, Szilvasi, A, Rajczy, K, Inotai, D, Gopcsa, L, Lengyel, L, Barta, A, Reti, M, Tordai, A, and Masszi, T

Subjects
*HLA-DR antigens, *HEMATOPOIETIC stem cell transplantation, *TREATMENT effectiveness, *THROMBOTIC microangiopathies, *THROMBOTIC thrombocytopenic purpura treatment, *GRAFT versus host disease, *PATIENTS
Abstract

Transplantation-associated thrombotic microangiopathy (TA-TMA) is a serious complication of allogeneic haematopoietic stem cell transplantation (allo-HSCT) with high mortality rate. We retrospectively studied the frequency, clinical and genetic associations and prognostic effect of TA-TMA, in a total of 425 consecutive adult patients, who underwent allo-HSCT for a malignant haematological condition between 2007 and 2013 at our single centre. TA-TMA developed in 19% of the patients. Unrelated donor type (P<0.001), acute GvHD grades II-IV (P<0.001), myeloablative conditioning regimens (P=0.003), tacrolimus-based GvHD prophylaxis (P=0.003), CMV infection (P=0.003) and carriership for HLA-DRB1*11 (P=0.034) were associated with the development of TA-TMA. Survival was adversely affected by the presence of TA-TMA (P<0.001). Among patients with TA-TMA, the outcome of HLA-DRB1*11 carriers was significantly better compared with non-carriers (P=0.003). As a new finding, our observations suggest that the presence of HLA-DRB1*11 antigen contributes to the development of TA-TMA and affects the outcome. [ABSTRACT FROM AUTHOR]

Published
2015
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