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1. Accuracy of physician assessment of treatment preferences and health status in elderly patients with higher-risk myelodysplastic syndromes

Author

Caocci, G., Voso, M.T., Angelucci, E., Stauder, R., Cottone, F., Abel, G., Nguyen, K., Platzbecker, U., Beyne-Rauzy, O., Gaidano, G., Invernizzi, R., Molica, S., Criscuolo, M., Breccia, M., Lübbert, M., Sanpaolo, G., Buccisano, F., Ricco, A., Palumbo, G.A., Niscola, P., Zhang, H., Fenu, S., La Nasa, G., Mandelli, F., and Efficace, F.

Published
2015
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8. PF644 CARFILZOMIB, LENALIDOMIDE AND DEXAMETHASONE IN RELAPSED/REFRACTORY MULTIPLE MYELOMA PATIENTS: THE REAL LIFE EXPERIENCE OF RETE EMATOLOGICA PUGLIESE (REP)

Author

Mele, A., primary, Prete, E., additional, De Risi, C., additional, Greco, G., additional, Citiso, S., additional, Cascavilla, N., additional, Falcone, A.P., additional, Sanpaolo, G., additional, Pastore, D., additional, Mele, G., additional, Giannotta, A., additional, Renzo, N. Di, additional, Vergine, C., additional, Reddiconto, G., additional, Mazza, P., additional, Palazzo, G., additional, Sabatelli, S., additional, Tarantini, G., additional, Germano, C., additional, Miccolis, R., additional, Specchia, G., additional, Curci, P., additional, Rizzi, R., additional, Guarini, A., additional, Capalbo, S., additional, Morciano, M.R., additional, Greco, A., additional, De Francesco, R., additional, Sibilla, S., additional, and Pavone, V., additional

Published
2019
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9. Accuracy of physician assessment of treatment preferences and health status in elderly patients with higher-risk myelodysplastic syndromes

Author

Caocci, G, Voso, Mt, Angelucci, E, Stauder, R, Cottone, F, Abel, G, Nguyen, K, Platzbecker, U, Beyne Rauzy, O, Gaidano, G, Invernizzi, R, Molica, S, Criscuolo, Marianna, Breccia, M, Lübbert, M, Sanpaolo, G, Buccisano, F, Ricco, A, Palumbo, Ga, Niscola, P, Zhang, H, Fenu, S, La Nasa, G, Mandelli, F, Efficace, F., Caocci, G, Voso, Mt, Angelucci, E, Stauder, R, Cottone, F, Abel, G, Nguyen, K, Platzbecker, U, Beyne Rauzy, O, Gaidano, G, Invernizzi, R, Molica, S, Criscuolo, Marianna, Breccia, M, Lübbert, M, Sanpaolo, G, Buccisano, F, Ricco, A, Palumbo, Ga, Niscola, P, Zhang, H, Fenu, S, La Nasa, G, Mandelli, F, and Efficace, F.

Abstract

Higher-risk myelodysplastic syndromes (MDS) are rarely curable and have a poor prognosis. We investigated the accuracy of physicians' perception of patients' health status and the patients' preferences for involvement in treatment decisions. We examined 280 newly diagnosed higher-risk elderly MDS patients paired with their physicians. Survey tools included the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) and the Control Preference Scale. Overall concordance was 49% for physician perception of patient preferences for involvement in treatment decisions. In 36.4% of comparisons there were minor differences and in 14.6% there were major differences. In 44.7% of the patients preferring a passive role, physicians perceived them as preferring an active or collaborative role. Absence of the patient's request for prognostic information (P=0.001) and judging the patient as having a poor health status (P=0.036) were factors independently associated with the physicians' attitude toward a lower degree of patient involvement in clinical decisions. Agreement on health status was found in 27.5% of cases. Physicians most frequently tended to overestimate health status of patients who reported low-level health status. The value of decision aid-tools in the challenging setting of higher-risk MDS should be investigated to further promote patient-centered care.

Published
2015

10. Prevalence, severity and correlates of fatigue in newly diagnosed patients with myelodysplastic syndromes.

Author

Efficace, F, Gaidano, G, Breccia, M, Criscuolo, Marianna, Cottone, F, Caocci, G, Bowen, D, Lubbert, M, Angelucci, E, Stauder, R, Selleslag, D, Platzbecker, U, Sanpaolo, G, Jonasova, A, Buccisano, F, Specchia, G, Palumbo, G. A, Niscola, P, Wan, C, Zhang, H, Fenu, S, Klimek, V, Beyne Rauzy, O, Nguyen, K, Mandelli, F., Efficace, F, Gaidano, G, Breccia, M, Criscuolo, Marianna, Cottone, F, Caocci, G, Bowen, D, Lubbert, M, Angelucci, E, Stauder, R, Selleslag, D, Platzbecker, U, Sanpaolo, G, Jonasova, A, Buccisano, F, Specchia, G, Palumbo, G. A, Niscola, P, Wan, C, Zhang, H, Fenu, S, Klimek, V, Beyne Rauzy, O, Nguyen, K, and Mandelli, F.

Abstract

The primary objective of this study was to investigate factors associated with fatigue severity in newly diagnosed patients with higher-risk myelodysplastic syndromes (MDS). The secondary objectives were to assess symptom prevalence and to examine the relationships between fatigue, quality of life (QoL) and overall symptom burden in these patients. The analyses were conducted in 280 higher-risk MDS patients. Pre-treatment patient-reported fatigue was evaluated with the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale and QoL was assessed with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). Female gender (P = 0·018), poor performance status (i.e., ECOG of 2-4) (P < 0·001) and lower levels of haemoglobin (Hb) (P = 0·026) were independently associated with higher fatigue severity. The three most prevalent symptoms were as follows: fatigue (92%), dyspnoea (63%) and pain (55%). Patients with higher levels of fatigue also had greater overall symptom burdens. The mean global QoL scores of patients with the highest versus those with the lowest levels of fatigue were 29·2 [standard deviation (SD), 18·3] and 69·0 (SD, 18·8), respectively and this difference was four times the magnitude of a clinically meaningful difference. Patient-reported fatigue severity revealed the effects of disease burden on overall QoL more accurately than did degree of anaemia. Special attention should be given to the female patients in the management of fatigue.

Published
2015

11. Involvement in treatment decisions, desire for prognostic information and quality of life in high-risk myelodysplastic syndromes: the physician's perspective

Author

Efficace, F, Alimena, G, Voso, Mt, Caocci, G, Di Tucci, A, Stauder, R, Sanpaolo, G, Gaidano, G, Breccia, M, Selleslag, D, Beyne-Rauzy, O, Wood, S, Ciceri, F, Specchia, G, Bowen, D, Buccisano, F, Deschler, B, Neuwirtova, R, Focan, C, Ackroyd, S, Criscuolo, M, Invernizzi, R, Ravoet, C, Paolini, R, Matta, G, Fenu, S, De Bock, R, Morra, E, Cottone, F, Vignetti, M, and Mandelli, F

Published
2011

15. Azacitidine for the treatment of lower risk myelodysplastic syndromes : aretrospective study of 74 patients enrolled in an Italian named patient program

Author

Musto, P, Maurillo, L, Spagnoli, A, Gozzini, A, Rivellini, F, Lunghi, M, Villani, O, Aloe Spiriti MA, Venditti, A, Santini, V, Collaborators: Leone G, Ad Hoc Italian Cooperative Study Group on Azacitidine in Myelodysplastic Syndromes Acute L. e. u. k. e. m. i. a. s., Voso, Mt, D'Arco, Am, Tatarelli, C, Ferrero, D, Gaidano, G, Palumbo, G, Di Raimondo, F, Oliva, E, Sanpaolo, G, Tonso, A, Santagostino, A, Filardi, N, Candoni, A, Fili, C, Russo, Domenico, Orciuolo, E, Petrini, M, Ciuffreda, L, Riezzo, A, Morabito, F, Mazza, P, Pastore, D, and Specchia, G.

Published
2010

16. Deferasirox for Transfusion-Dependent Patients with Myelodysplastic Syndromes: Safety, Efficacy, and Beyond (GIMEMA MDS0306 Trial)

Author

Angelucci, E, Santini, V, Tucci, Aad, Quaresmini, G, Finelli, C, Volpe, A, Quarta, G, Rivellini, F, Sanpaolo, G, Cilloni, D, Salvi, F, Caocci, G, Molteni, A, Vallisa, D, Voso, Maria Teresa, Fenu, S, Borin, L, Latte, G, Alimena, G, Storti, Sergio, Piciocchi, A, Fazi, P, Vignetti, M, Tura, S., Storti, S (ORCID:0000-0002-4374-3985), Angelucci, E, Santini, V, Tucci, Aad, Quaresmini, G, Finelli, C, Volpe, A, Quarta, G, Rivellini, F, Sanpaolo, G, Cilloni, D, Salvi, F, Caocci, G, Molteni, A, Vallisa, D, Voso, Maria Teresa, Fenu, S, Borin, L, Latte, G, Alimena, G, Storti, Sergio, Piciocchi, A, Fazi, P, Vignetti, M, Tura, S., and Storti, S (ORCID:0000-0002-4374-3985)

Abstract

In the absence of randomized, controlled trial data to support iron chelation therapy in transfusion-dependent patients with myelodysplastic syndromes (MDS), continued evidence from large prospective clinical trials evaluating the efficacy and safety of iron chelation therapy in this patient population is warranted.

Published
2014

17. Preference for involvement in treatment decisions and request for prognostic information in newly diagnosed patients with higher-risk myelodysplastic syndromes

Author

Efficace, F, Gaidano, G, Sprangers, M, Cottone, F, Breccia, M, Voso, Maria Teresa, Caocci, G, Stauder, R, Di Tucci, Aa, Sanpaolo, G, Selleslag, D, Angelucci, E, Platzbecker, U, Mandelli, F., Efficace, F, Gaidano, G, Sprangers, M, Cottone, F, Breccia, M, Voso, Maria Teresa, Caocci, G, Stauder, R, Di Tucci, Aa, Sanpaolo, G, Selleslag, D, Angelucci, E, Platzbecker, U, and Mandelli, F.

Abstract

The main objective of this study was to assess preferences for involvement in treatment decisions and requests for prognostic information in newly diagnosed higher-risk myelodysplastic syndrome (MDS) patients.

Published
2014

19. 5-AZACYTIDINE FOR THE TREATMENT OF INTERMEDIATE-2/HIGH IPSS RISK MYELODYSPLASTIC SYNDROMES: RESULTS IN 83 PATIENTS FROM THE ITALIAN PATIENT NAMED PROGRAM

Author

Maurillo, L., Alessandra Spagnoli, Gozzini, A., Cecconi, N., D Argenio, M., Lunghi, M., Rocco, S., Palumbo, G., Rivellini, E., Genuardi, M., Sibilla, S., Ferrara, F., Mele, G., Filardi, N., Sanpaolo, G., Specchia, G., Tonso, A., Santagostino, A., Voso, M. T., Balleari, E., Cassibba, V., Della Cioppa, P., Mazzone, C., Oliva, E., Ciuffreda, L., Russo, D., Galimberti, S., Villani, O., D Auria, F., Di Renzo, N., and D Arco, A. M.

Published
2008

20. 5-Azacytidine in 82 Low/Intermediate-1 IPSS Risk Myelodysplastic Syndromes: Results from the Italian Patient Named Program

Author

Musto, L. M., Spagnoli, Alessandra, Gozzini, A., Rivellini, F., Tatarelli, C., Lunghi, M., Fili, C., Orciuolo, E., Ciuffreda, L., Vigna, E., Cioppa, P. D., Candoni, A., Ferrero, D., Palmieri, S., Palumbo, G., Renzo, N. D., Oliva, E., Sanpaolo, G., Pastore, D., Tonso, A., Santagostino, A., Rocco, S., Villani, O., D'Auria, F., D'Arco, A. M., Gaidano, G., Galimberti, S., Russo, D., Venditti, A., Aloe Spiriti, M. A., Leone, G., and Santini, V.

Published
2008

21. 5-azacytidine For the Treatment of Low/intermediate-1 Ipss Risk Myelodysplastic Syndromes: Results In 63 Patients From the Italian Patient Named Program

Author

Musto, P., Maurillo, L., Alessandra Spagnoli, Gozzini, A., Rivellini, F., Tatarelli, C., Lunghi, M., Fili, C., Orciuolo, E., Ciuffreda, L., Vigna, E., Della Cioppa, P., Ferrero, D., Palmieri, S., Palumbo, G., Di Renzo, N., Oliva, E., Sanpaolo, G., Pastore, D., Tonso, A., Santagostino, A., Villani, O., D Auria, F., D Arco, A., Gaidano, G., Galimberti, S., Russo, D., Venditti, A., Aloe-Spiriti, M. A., Leone, G., and Santini, V.

Published
2008

22. 5-AZACYTIDINE FOR THE TREATMENT OF LOW/INTERMEDIATE-1 IPSS RISK MYELODYSPLASTIC SYNDROMES : RESULTS IN 63 PATIENTS FROM THE ITALIAN PATIENT NAMED PROGRAM

Author

Mustop, Maurillo, L, Spagnoli, A, Gozzini, A, Rivellini, F, Tatarelli, F, Lunghi, M, Fili, C, Orciuolo, E, Ciuffreda, L, Vigna, E, Della Cioppa, D, Ferrero, D, Palmieri, S, Palumbo, G, Di Renzo, N, Oliva, E, Sanpaolo, G, Pastore, A, Tonso, A, Villani O, Santagostino A., D'Auria, F, D'Arco, A, Gaidano, G, Galimberti, Sara, Russo, D, Venditti, A, Aloe Spiriti MA, Leone, G, and Santini, V.

Published
2008

23. Advanced mast cell disease: an Italian Hematological Multicenter experience

Author

Pagano, L, Valentini, C, Caira, M, Rondoni, M, Van Lint, M, Candoni, A, Allione, B, Cattaneo, C, Marbello, L, Caramatti, C, Pogliani, E, Iannitto, E, Giona, F, Ferrara, F, Invernizzi, R, Fanci, R, Lunghi, M, Fianchi, L, Sanpaolo, G, Stefani, P, Pulsoni, A, Martinelli, G, Leone, G, Musto, P, Valentini, CG, Van Lint, MT, Stefani, PM, Musto, P., POGLIANI, ENRICO MARIA, Pagano, L, Valentini, C, Caira, M, Rondoni, M, Van Lint, M, Candoni, A, Allione, B, Cattaneo, C, Marbello, L, Caramatti, C, Pogliani, E, Iannitto, E, Giona, F, Ferrara, F, Invernizzi, R, Fanci, R, Lunghi, M, Fianchi, L, Sanpaolo, G, Stefani, P, Pulsoni, A, Martinelli, G, Leone, G, Musto, P, Valentini, CG, Van Lint, MT, Stefani, PM, Musto, P., and POGLIANI, ENRICO MARIA

Abstract

The aim of the study is to evaluate clinical features, treatments and outcome of patients with systemic mast cell disease (MCD) who arrived to the attention of hematologists. A retrospective study was conducted over 1995-2006 in patients admitted in 18 Italian hematological divisions. Twenty-four cases of advanced MCD were collected: 12 aggressive SM (50%), 8 mast cell leukemia (33%), 4 SM with associated clonal non-mast cell-lineage hematologic disease (17%). Spleen and liver were the principal extramedullary organ involved. The c-kit point mutation D816V was found in 13/18 patients in which molecular biology studies were performed (72%). Treatments were very heterogeneous: on the whole Imatinib was administered in 17 patients, alpha-Interferon in 8, 2-CdA in 3; 2 patients underwent allogeneic hematopoietic stem cell transplantation. The overall response rate to Imatinib, the most frequently employed drugs, was of 29%, registering one complete remission and four partial remission; all responsive patients did not present D816V c-kit mutation. Overall three patients (12%) died for progression of disease. We conclude that MCD is characterized by severe mediator-related symptoms but with a moderate mortality rate. D816V c-kit mutation is frequent and associated with resistance against Imatinib. Because of the rarity of these forms, an effective standard of care is lacking. More data are needed to find new and successful therapeutic strategies.

Published
2008

24. P-310 Eltrombopag for the treatment of thrombocytopenia of low and intermediate-1 IPSS risk myelodysplastic syndromes: Results of a prospective, randomized trial

Author

Oliva, E.N., primary, Santini, V., additional, Zini, G., additional, Palumbo, G.A., additional, Poloni, A., additional, Cortelezzi, A., additional, Rodeghiero, F., additional, Voso, M.T., additional, Molteni, A., additional, Sanpaolo, G., additional, Liberati, M., additional, Morabito, F., additional, Balleari, E., additional, Impera, S., additional, Salvi, F., additional, Spiriti, M.A. Aloe, additional, Marino, A., additional, Rodà, F., additional, Alati, C., additional, Ronco, F., additional, Di Raimondo, F., additional, Leoni, P., additional, Alimena, G., additional, Latagliata, R., additional, and Nobile, F., additional

Published
2013
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25. Peg-filgrastim versus filgrastim after autologous stem cell tranplantation: case-control study in patients with multiple myeloma and review of the literature

Author

Musto, P, Scalzulli, P, Terruzzi, E, Rossini, F, Iacopino, P, Messina, G, Guariglia, R, Pietrantuono, G, Villani, O, D'Auria, F, Falcone, A, Sanpaolo, G, Valvano, M, Pogliani, E, Morabito, F, Scalzulli, PR, Valvano, MR, Morabito, F., POGLIANI, ENRICO MARIA, Musto, P, Scalzulli, P, Terruzzi, E, Rossini, F, Iacopino, P, Messina, G, Guariglia, R, Pietrantuono, G, Villani, O, D'Auria, F, Falcone, A, Sanpaolo, G, Valvano, M, Pogliani, E, Morabito, F, Scalzulli, PR, Valvano, MR, Morabito, F., and POGLIANI, ENRICO MARIA

Abstract

We investigated the effects of a single s.c. injection of peg-filgrastim in 32 patients with multiple myeloma who underwent autologous stem cell transplantation (AuSCT) as first line treatment. For comparison, 32 myeloma patients with similar characteristics and receiving standard daily administration of filgrastim were matched. Overall, there were no statistically significant differences between peg-filgrastim and filgrastim in terms of tolerability, marrow recovery, severity of neutropenia, incidence and duration of febrile neutropenia, documented infections and transfusions. However, some favourable trends or effects in favour of peg-filgrastim were observed. This was confirmed by a review of the published papers about this topic.

Published
2007

28. 147 Involvement in treatment decisions, desire for prognostic information and quality of life in high-risk myelodysplastic syndromes: the physician's perspective

Author

Efficace, F., primary, Alimena, G., additional, Voso, M.T., additional, Caocci, G., additional, Di Tucci, A., additional, Stauder, R., additional, Sanpaolo, G., additional, Gaidano, G., additional, Breccia, M., additional, Selleslag, D., additional, Beyne-Rauzy, O., additional, Wood, S., additional, Ciceri, F., additional, Specchia, G., additional, Bowen, D., additional, Buccisano, F., additional, Deschler, B., additional, Neuwirtova, R., additional, Focan, C., additional, Ackroyd, S., additional, Criscuolo, M., additional, Invernizzi, R., additional, Ravoet, C., additional, Paolini, R., additional, Matta, G., additional, Fenu, S., additional, De Bock, R., additional, Morra, E., additional, Cottone, F., additional, Vignetti, M., additional, and Mandelli, F., additional

Published
2011
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29. 98 Clinically meaningful responses in low risk MDS patients with del5q treated with lenalidomide: Quality of life and cardiac changes

Author

Oliva, E., primary, Latagliata, R., additional, Spiriti, M.A. Aloe, additional, Cortelezzi, A., additional, Balleari, E., additional, Poloni, A., additional, Sanpaolo, G., additional, Finelli, C., additional, Volpe, A., additional, Ricco, A., additional, Palumbo, G., additional, Baratè, C., additional, Ronco, F., additional, Morabito, F., additional, Alati, C., additional, Breccia, M., additional, Alimena, G., additional, and Nobile, F., additional

Published
2011
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33. Intermediate Dose Melphalan, Bortezomib, Thalidomide, Dexametasone (MVTD) Conditioning Therapy and ASCT in Relapsed Multiple Myeloma Patients: A Single Center Experience.

Author

Scalzulli, Potito Rosario, primary, Valvano, Maria Rosa, additional, Bodenizza, Carlo C., additional, Carella, Angelo Michele, additional, Dell’Olio, Matteo M., additional, Falcone, Antonietta A.P., additional, Greco, Michele Mario, additional, La Sala, Antonio A., additional, Mantuano, Francesco F.S., additional, Melillo, Lorella L., additional, Merla, Emanuela E., additional, Nobile, Michele M., additional, Sanpaolo, Sanpaolo G., additional, and Cascavilla, Nicola N., additional

Published
2007
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35. Intermediate Dose Melphalan, Bortezomib, Thalidomide, Dexametasone (MVTD) Conditioning Therapy and ASCT in Relapsed Multiple Myeloma Patients: A Single Center Experience

Author

Sanpaolo G. Sanpaolo, Potito Rosario Scalzulli, Francesco Saverio Mantuano, Antonio La Sala, Nicola Cascavilla, Maria Rosa Valvano, Antonietta Falcone, Matteo Dell’Olio, Michele Nobile, Michele Mario Greco, Carlo C. Bodenizza, Lorella Melillo, Angelo Michele Carella, and Emanuela Merla

Subjects
Melphalan, medicine.medical_specialty, Bortezomib, business.industry, Immunology, Cell Biology, Hematology, Single Center, medicine.disease, Biochemistry, Chemotherapy regimen, Gastroenterology, Surgery, Thalidomide, Internal medicine, medicine, Autologous transplantation, Progression-free survival, business, Multiple myeloma, medicine.drug
Abstract

Background: Autologous Transplantation with intermediate or high doses of Melphalan is the most effective therapy in patients with Multiple Myeloma being < 65 years old, although this procedure is limited by an high percentage of relapses. Recent advances knowledge of the molecular mechanisms, allowed the development of novel targeted therapies. Several studies have shown that the synergic effect of Bortezomib and Talidomidomide, in association with conventional chemotherapies, induces a more pronounced reduction of tumour mass, an enhancement of global responses and an improvement of Progression Free Survival (PFS). Aims: The present study is aimed at the evaluation of effectiveness and toxicity of MVTD conditioning therapy with stem cell support in Multiple Myeloma relapsed patients. Patients and Methods: From January 2005 to August 2007, 15 patients (7 males and 8 females: median age 62 yrs (46–70)) have been subjected to Autologous Transplantation therapy with MVTD conditioning therapy (Melphalan 100 mg/sqm days - 6 and - 3, Bortezomib 1.3 mg/sqm days - 6 and - 3, Talidomide 200 mg from day - 6 to day - 2, Desametazone from day - 6 to day - 3, CSP infusion at day 0). All patients were in CS III A; median performed therapies 3 (1–5); all had received previously a therapy with Talidomide, two Talidomide + Velcade, one PEG-IFN. Results: 100% of patients obtained a response: 4 patients (26.7%) obtained CR, 5 (33.3%) a nCR, 4 (26.7%) a VGPR and 2 (13.3%) a PR. One patient in CR was subjected to 2 therapeutic lines and 3 patients in CR were subjected to more than 1 therapeutic lines, 5 in nCR from 2 to 4, 4 in VGPR from 2 to 4 and 2 in PR more than 2. At the moment, with a median of 7.5 months (1–21), 11 (73%) are alive, 8 (53%) still keep the obtained response, 3 (20%) after progression are in rescue therapy, 4 patients (27%) died because of progression. PFS median since MVTD beginning is of 6 months (1–24). None of the patients have developed a significant neurotoxicity. All patients have developed grade 4 thrombocytopenia and neutropoenia, with a median of 3 days (1–10) and 6 days (4–12), respectively. 54% of patients had neutropoenia with fever with a median of 2 days (0–8). The median units of platelets and red cells infused was 3 (2–11) and 2 (0–11), respectively. Conclusions: In our experience, Autologous Transplantation therapy with MVTD conditioning, appeared to be effective in relapsed patients with Multiple Myeloma. Although in a small series of patients the response obtained was independent from the number and type of previous therapies, from the clinical state, and β2 microglobulin. No significant neurotoxicity has been observed, whether the haematological toxicity was overlapping to that due to conventional Autologous Transplant. Further studies, in larger series of non pluritreated patients are required.

Published
2007

41. Systemic mastocytosis. A Gimema multicentric survey

Author

Pagano, L., Valentini, C. G., Caira, M., Musto, F., Rondoni, M., Lint, M. T., anna candoni, Martinelli, G., Gatto, S., Cattaneo, C., Marbello, L., Caramatti, C., Castagnola, C., Pogliani, E. M., Mitra, M. E., Giona, F., Ferrara, F., Invernizzi, R., Fanci, R., Lunghi, M., Rossi, G., Fianchi, L., Sanpaolo, G., Pulsoni, A., and Leone, G.

46. Real-world efficacy and safety of luspatercept and predictive factors of response in patients with lower risk myelodysplastic syndromes with ring sideroblasts

Author

Lanino, L., Restuccia, F., Perego, A., Ubezio, M., Fattizzo, B., Riva, M., Consagra, A., Musto, P., Cilloni, D., Oliva, E. N., Palmieri, R., Poloni, A., Califano, C., Capodanno, I., Itri, F., Elena, C., Fozza, C., Pane, F., Pelizzari, A. M., Breccia, M., Di Bassiano, F., Crisa, E., Ferrero, D., Giai, V., Barraco, D., Vaccarino, A., Griguolo, D., Minetto, P., Quintini, M., Paolini, S., Sanpaolo, G., Sessa, M., Bocchia, M., Di Renzo, N., Diral, E., Leuzzi, L., Genua, A., Guarini, A., Molteni, A., Nicolino, B., Occhini, U., Rivoli, G., Bono, R., Calvisi, A., Castelli, A., Di Bona, E., Di Veroli, A., Ferrara, F., Fianchi, L., Galimberti, S., Grimaldi, D., Marchetti, M., Norata, M., Frigeni, M., Sancetta, R., Selleri, C., Tanasi, I., Tosi, P., Turrini, M., Giordano, L., Finelli, C., Pasini, P., Naldi, I., Santini, V., and Della Porta, M. G.

47. Advanced mast cell disease: an Italian Hematological Multicenter experience

Author

Monia Lunghi, Pietro Maria Stefani, Anna Candoni, Laura Marbello, Bernardino Allione, Alessandro Pulsoni, Luana Fianchi, Michela Rondoni, Fiorina Giona, Livio Pagano, Pellegrino Musto, Rosangela Invernizzi, Caterina Giovanna Valentini, Cecilia Caramatti, Chiara Cattaneo, Giovanni Martinelli, Emilio Iannitto, Grazia Sanpaolo, Enrico Pogliani, Giuseppe Leone, Rosa Fanci, Morena Caira, Felicetto Ferrara, Maria Teresa Van Lint, Pagano, L, Valentini, C, Caira, M, Rondoni, M, Van Lint, M, Candoni, A, Allione, B, Cattaneo, C, Marbello, L, Caramatti, C, Pogliani, E, Iannitto, E, Giona, F, Ferrara, F, Invernizzi, R, Fanci, R, Lunghi, M, Fianchi, L, Sanpaolo, G, Stefani, P, Pulsoni, A, Martinelli, G, Leone, G, Musto, P, Pagano L, Valentini CG, Caira M, Rondoni M, Van Lint MT, Candoni A, Allione B, Cattaneo C, Marbello L, Caramatti C, Pogliani EM, Iannitto E, Giona F, Ferrara F, Invernizzi R, Fanci R, Lunghi M, Fianchi L, Sanpaolo G, Stefani PM, Pulsoni A, Martinelli G, Leone G, and Musto P.

Subjects
Male, medicine.medical_treatment, Hematopoietic stem cell transplantation, Piperazines, Antineoplastic Agent, Retrospective Studie, MED/15 - MALATTIE DEL SANGUE, tyrosine kinase inhibitors, Transplantation, Homologou, Hematology, Mastocytosi, Remission Induction, Hematopoietic Stem Cell Transplantation, Middle Aged, Mast cell leukemia, Survival Rate, Proto-Oncogene Proteins c-kit, Hematologic disease, Italy, Liver, Benzamides, Imatinib Mesylate, Female, Mastocytosis, medicine.drug, Human, Adult, medicine.medical_specialty, Mast cell disease, Therapy, Tyrosine kinase inhibitors, Antineoplastic Agents, Antiviral Agents, SYSTEMIC MAST CELL DISEASE (MCD), Disease-Free Survival, Internal medicine, medicine, Humans, Point Mutation, Transplantation, Homologous, Survival rate, Piperazine, Aged, Retrospective Studies, Antiviral Agent, therapy, business.industry, mast cell disease, Interferon-alpha, Imatinib, medicine.disease, Transplantation, Imatinib mesylate, Pyrimidines, Pyrimidine, Amino Acid Substitution, Immunology, business, Spleen
Abstract

The aim of the study is to evaluate clinical features, treatments and outcome of patients with systemic mast cell disease (MCD) who arrived to the attention of hematologists. A retrospective study was conducted over 1995-2006 in patients admitted in 18 Italian hematological divisions. Twenty-four cases of advanced MCD were collected: 12 aggressive SM (50%), 8 mast cell leukemia (33%), 4 SM with associated clonal non-mast cell-lineage hematologic disease (17%). Spleen and liver were the principal extramedullary organ involved. The c-kit point mutation D816V was found in 13/18 patients in which molecular biology studies were performed (72%). Treatments were very heterogeneous: on the whole Imatinib was administered in 17 patients, alpha-Interferon in 8, 2-CdA in 3; 2 patients underwent allogeneic hematopoietic stem cell transplantation. The overall response rate to Imatinib, the most frequently employed drugs, was of 29%, registering one complete remission and four partial remission; all responsive patients did not present D816V c-kit mutation. Overall three patients (12%) died for progression of disease. We conclude that MCD is characterized by severe mediator-related symptoms but with a moderate mortality rate. D816V c-kit mutation is frequent and associated with resistance against Imatinib. Because of the rarity of these forms, an effective standard of care is lacking. More data are needed to find new and successful therapeutic strategies.

Published
2008

48. Prognostic Factors for Overall Survival In Chronic Myeloid Leukemia Patients: A Multicentric Cohort Study by the Italian CML GIMEMA Network

Author

Giovanni Reddiconto, Maria Stella De Candia, Sara Galimberti, Fabrizio Pane, Michele Pizzuti, Luciano Levato, Massimiliano Bonifacio, Massimo Breccia, Anna Rita Scortechini, Elisabetta Abruzzese, Giovanni Caocci, Valentina Giai, Paolo Ditonno, Andrea Patriarca, Federica Sorà, Giuseppe Pietrantuono, Gaetano La Barba, Fabio Stagno, Patrizia Pregno, Antonella Gozzini, Francesco Di Raimondo, Michele Baccarani, Grazia Sanpaolo, Giovannino Ciccone, Micaela Bergamaschi, Gianni Binotto, Alessandro Maggi, Chiara Monagheddu, Giuseppe Saglio, Maria Cristina Miggiano, Monica Bocchia, Pellegrino Musto, Anna Guella, Luigiana Luciano, Giorgina Specchia, Bruno Martino, Francesco Albano, Fabio Saccona, Giovanna Rege-Cambrin, Olga Mulas, Gianantonio Rosti, Carmen Fava, Mario Tiribelli, Fausto Castagnetti, Isabella Capodanno, Specchia G., Pregno P., Breccia M., Castagnetti F., Monagheddu C., Bonifacio M., Tiribelli M., Stagno F., Caocci G., Martino B., Luciano L., Pizzuti M., Gozzini A., Scortechini A.R., Albano F., Bergamaschi M., Capodanno I., Patriarca A., Fava C., Rege-Cambrin G., Sora F., Galimberti S., Bocchia M., Binotto G., Reddiconto G., DiTonno P., Maggi A., Sanpaolo G., De Candia M.S., Giai V., Abruzzese E., Miggiano M.C., La Barba G., Pietrantuono G., Guella A., Levato L., Mulas O., Saccona F., Rosti G., Musto P., Di Raimondo F., Pane F., Baccarani M., Saglio G., and Ciccone G.

Subjects
ELT, chronic myeloid leukemia, ELTs, prognostic factors, Sokal score, tyrosine kinase inhibitors, Cancer Research, medicine.medical_specialty, Patient characteristics, Internal medicine, Overall survival, Medicine, Prospective cohort study, prognostic factor, RC254-282, Original Research, business.industry, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, Myeloid leukemia, Imatinib, prognostic factors for overall survival in chronic myeloid leukemia patient a multicentric cohort study, Settore MED/15 - MALATTIE DEL SANGUE, Oncology, Cohort, business, Sokal Score, Cohort study, medicine.drug
Abstract

An observational prospective study was conducted by the CML Italian network to analyze the role of baseline patient characteristics and first line treatments on overall survival and CML-related mortality in 1206 newly diagnosed CML patients, 608 treated with imatinib (IMA) and 598 with 2nd generation tyrosine kinase inhibitors (2GTKI). IMA-treated patients were much older (median age 69 years, IQR 58-77) than the 2GTKI group (52, IQR 41-63) and had more comorbidities. Estimated 4-year overall survival of the entire cohort was 89% (95%CI 85.9-91.4). Overall, 73 patients (6.1%) died: 17 (2.8%) in the 2GTKI vs 56 (9.2%) in the IMA cohort (adjusted HR=0.50; 95% CI=0.26-0.94), but no differences were detected for CML-related mortality (10 (1.7%) vs 11 (1.8%) in the 2GTKIs vs IMA cohort (sHR=1.61; 0.52-4.96). The ELTS score was associated to CML mortality (high risk vs low, HR=9.67; 95%CI 2.94-31.74; p

Published
2021

49. Peg-filgrastim versus filgrastim after autologous stem cell tranplantation: case-control study in patients with multiple myeloma and review of the literature

Author

Pellegrino Musto, Giuseppe Messina, Fortunato Morabito, Potito Rosario Scalzulli, Grazia Sanpaolo, Maria Rosa Valvano, Fiorella D'Auria, Antonietta Falcone, Roberto Guariglia, Giuseppe Pietrantuono, Elisabetta Terruzzi, Fausto Rossini, Oreste Villani, Enrico Pogliani, Pasquale Iacopino, Musto, P, Scalzulli, P, Terruzzi, E, Rossini, F, Iacopino, P, Messina, G, Guariglia, R, Pietrantuono, G, Villani, O, D'Auria, F, Falcone, A, Sanpaolo, G, Valvano, M, Pogliani, E, and Morabito, F

Subjects
Oncology, Cancer Research, medicine.medical_specialty, Filgrastim, Neutropenia, Polyethylene Glycol, Polyethylene Glycols, Autologous stem-cell transplantation, MED/15 - MALATTIE DEL SANGUE, Internal medicine, Granulocyte Colony-Stimulating Factor, medicine, Humans, Autologous transplantation, Multiple myeloma, business.industry, Hematology, medicine.disease, Recombinant Proteins, Surgery, Granulocyte colony-stimulating factor, Treatment Outcome, Tolerability, Case-Control Studies, business, Multiple Myeloma, Febrile neutropenia, medicine.drug, Stem Cell Transplantation, Human
Abstract

We investigated the effects of a single s.c. injection of peg-filgrastim in 32 patients with multiple myeloma who underwent autologous stem cell transplantation (AuSCT) as first line treatment. For comparison, 32 myeloma patients with similar characteristics and receiving standard daily administration of filgrastim were matched. Overall, there were no statistically significant differences between peg-filgrastim and filgrastim in terms of tolerability, marrow recovery, severity of neutropenia, incidence and duration of febrile neutropenia, documented infections and transfusions. However, some favourable trends or effects in favour of peg-filgrastim were observed. This was confirmed by a review of the published papers about this topic.

Published
2007

50. Real-world efficacy and safety of luspatercept and predictive factors of response in patients with lower risk myelodysplastic syndromes with ring sideroblasts.

Author

Lanino L, Restuccia F, Perego A, Ubezio M, Fattizzo B, Riva M, Consagra A, Musto P, Cilloni D, Oliva EN, Palmieri R, Poloni A, Califano C, Capodanno I, Itri F, Elena C, Fozza C, Pane F, Pelizzari AM, Breccia M, Di Bassiano F, Crisà E, Ferrero D, Giai V, Barraco D, Vaccarino A, Griguolo D, Minetto P, Quintini M, Paolini S, Sanpaolo G, Sessa M, Bocchia M, Di Renzo N, Diral E, Leuzzi L, Genua A, Guarini A, Molteni A, Nicolino B, Occhini U, Rivoli G, Bono R, Calvisi A, Castelli A, Di Bona E, Di Veroli A, Ferrara F, Fianchi L, Galimberti S, Grimaldi D, Marchetti M, Norata M, Frigeni M, Sancetta R, Selleri C, Tanasi I, Tosi P, Turrini M, Giordano L, Finelli C, Pasini P, Naldi I, Santini V, and Della Porta MG

Subjects
Humans, Activin Receptors, Type II, Immunoglobulin Fc Fragments, Myelodysplastic Syndromes drug therapy, Anemia, Sideroblastic drug therapy
Published
2023
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