Publications by authors named "Alessandra Pompa"

25 Publications

  • Page 1 of 1

Dose/Schedule-Adjusted Rd-R vs Continuous Rd for elderly, intermediate-fit, newly diagnosed multiple myeloma patients.

Blood 2021 Mar 19. Epub 2021 Mar 19.

University of Torino, Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino, Torino, Italy.

Lenalidomide-dexamethasone (Rd) is a standard treatment for elderly multiple myeloma (MM) patients. In this randomized, phase III study, we investigated the efficacy and feasibility of a dose/schedule-adjusted Rd followed by maintenance 10 mg/day without dexamethasone (Rd-R) vs continuous Rd in elderly, intermediate-fit newly diagnosed MM patients. The primary endpoint was event-free survival (EFS), defined as progression/death for any cause, lenalidomide discontinuation, any hematologic grade 4 or non-hematologic grade 3-4 adverse events (AEs). Of the 199 evaluable patients, 101 received Rd-R and 98 continuous Rd. Median follow-up was 37 months. Best response rates were comparable: ≥ partial response rates were 78% vs 68% (p=0.15) in Rd-R vs continuous Rd groups. EFS was 10.4 with Rd-R vs 6.9 months with continuous Rd (HR 0.70, 95% CI 0.51-0.95, p=0.02). Median progression-free survival was 20.2 vs 18.3 months (HR 0.78, 95% CI 0.55-1.10, p=0.16), 3-year overall survival was 74% vs 63% (HR 0.62, 95% CI 0.37-1.03, p=0.06). At least 1 non-hematologic grade ≥3 AE rate was 33% vs 43% (p=0.14); the most frequent grade ≥3 AEs were neutropenia (21% vs 18%), infections (10% vs 12%) skin disorders (7% vs 3%) in Rd-R vs Rd; constitutional and central nervous system AEs mainly related to dexamethasone were more frequent with continuous Rd. Lenalidomide was discontinued for AEs in 24% vs 30% and was reduced in 45% vs 62% of patients, in Rd-R vs Rd, respectively. In intermediate-fit patients, switching to reduced-dose lenalidomide maintenance without dexamethasone after 9 cycles of Rd was feasible, with similar outcome to standard continuous Rd.
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http://dx.doi.org/10.1182/blood.2020009507DOI Listing
March 2021

Application of Next-Generation Sequencing for the Genomic Characterization of Patients with Smoldering Myeloma.

Cancers (Basel) 2020 May 23;12(5). Epub 2020 May 23.

Department of Oncology and Hemato-oncology, University of Milan, 20122 Milan, Italy.

Genomic analysis could contribute to a better understanding of the biological determinants of the evolution of multiple myeloma (MM) precursor disease and an improved definition of high-risk patients. To assess the feasibility and value of next-generation sequencing approaches in an asymptomatic setting, we performed a targeted gene mutation analysis and a genome-wide assessment of copy number alterations (CNAs) by ultra-low-pass whole genome sequencing (ULP-WGS) in six patients with monoclonal gammopathy of undetermined significance and 25 patients with smoldering MM (SMM). Our comprehensive genomic characterization highlighted heterogeneous but substantial values of the tumor fraction, especially in SMM; a rather high degree of genomic complexity, in terms of both mutations and CNAs, and inter-patient variability; a higher incidence of gene mutations and CNAs in SMM, confirming ongoing evolution; intraclonal heterogeneity; and instances of convergent evolution. ULP-WGS of these patients proved effective in revealing the marked genome-wide level of their CNAs, most of which are not routinely investigated. Finally, the analysis of our small SMM cohort suggested that chr(8p) deletions, the DNA tumor fraction, and the number of alterations may have clinical relevance in the progression to overt MM. Although validation in larger series is mandatory, these findings highlight the promising impact of genomic approaches in the clinical management of SMM.
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http://dx.doi.org/10.3390/cancers12051332DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7281620PMC
May 2020

Smoldering multiple myeloma: the role of different scoring systems in identifying high-risk patients in real-life practice.

Leuk Lymphoma 2019 12 6;60(12):2968-2974. Epub 2019 Jun 6.

Ospedale Nuovo di Legnano, Division of Hematology, Legnano, Italy.

We explore the predictive role of 2014-updated International Myeloma Working Group (IMWG) diagnostic criteria and of some of currently available risk models for progression to symptomatic myeloma when applied in our unselected population of 75 smoldering multiple myeloma (SMM) patients observed between 2000 and 2015. Risk scores including routinely used clinical parameters such as bone marrow plasmacell infiltration rate, immunoparesis, serum monoclonal component (sMC) value, and altered free light chain ratio (FLCr), were clinically useful to identify SMM patients at higher risk of progression. Time to myeloma progression in our ultra-high risk SMM according to IMWG diagnostic update criteria was very short (12.4 months). Our analysis identified as independent reliable predictors of progression altered FLCr as well as increasing plasma cell infiltration which are part of most commonly applied risk models. Waiting for new scoring systems, bone marrow evaluation and complete laboratory screening are still milestones for SMM management.
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http://dx.doi.org/10.1080/10428194.2019.1620948DOI Listing
December 2019

Vestibular rehabilitation has positive effects on balance, fatigue and activities of daily living in highly disabled multiple sclerosis people: A preliminary randomized controlled trial.

Restor Neurol Neurosci 2018 ;36(6):709-718

Multiple Sclerosis Unit, IRCCS Santa Lucia Foundation, Rome, Italy.

Background: Balance disorders are present in patients with Multiple Sclerosis and part of these disorders could be of vestibular origin. Vestibular Rehabilitation was found to be effective in improving balance in patients with central vestibular dysfunction.

Objective: To investigate the clinical effects of vestibular rehabilitation on balance skills and secondly on fatigue and activity of daily living in highly disabled multiple sclerosis people.

Methods: Thirty hospitalized participants with severe multiple sclerosis (EDSS 6-7) were randomly assigned to the experimental group (15 patients -9F-; mean age 50.64±11.73) and the control group (15 patients -8F-; mean age 45.77±10.91). All patients were evaluated before and after treatment with the Expanded Disability Status Scale, Barthel Index Tinetti Balance and Gait scale, Berg Balance Scale, Fatigue Severity Scale, Two Minute Walking Test and Timed 25-foot walk test. Two follow-ups (i.e., at 30 and 60 days after treatment) were carried out with Barthel Index.

Results: Significant improvement was found in the experimental group with respect to the control group (p <  0,05) in balance, fatigue perception, activities of daily living and short distance gait. No significant improvements were found for gait endurance as measured by Two Minute Walking Test.

Conclusions: Four weeks of Vestibular Rehabilitation training results in less fatigue, improved balance and performance of the activities of daily living in patients with severe Multiple Sclerosis.
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http://dx.doi.org/10.3233/RNN-180850DOI Listing
April 2019

Lamivudine prophylaxis prevents hepatitis B virus reactivation in anti-HBc positive patients under rituximab for non-Hodgkin lymphoma.

Dig Liver Dis 2019 03 7;51(3):419-424. Epub 2018 Sep 7.

CRC "A. M. and A. Migliavacca" Center for Liver Disease, Division of Gastroenterology and Hepatology, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy. Electronic address:

Backgound: A significant proportion of hepatitis B surface antigen (HBsAg) negative/anti-hepatitis B core antigen (anti-HBc) positive patients with non-Hodgkin lymphoma (NHL) undergoing rituximab-based chemotherapy (R-CT) may suffer hepatitis B virus (HBV) reactivation.

Aims: We wanted to assess efficacy and safety of lamivudine (LMV) prophylaxis to prevent this complication.

Methods: Eighty-five consecutive HBsAg negative/anti-HBc positive NHL patients (71 years, 100% serum HBV DNA undetectable, 74% anti-HBs positive) received LMV coadministered with R-CT and for 18 months after the end of R-CT. Serum ALT, HBsAg, anti-HBs and HBV DNA were assessed every 4 months during and after end of LMV.

Results: During 39 (2-108) months of study period, including 21 months of LMV and 27 additional months after LMV discontinuation, one patient (2%) had HBV reactivation, 31 months after stopping LMV and during administration of new immunosuppressive regimens, without LMV prophylaxis, owing to incomplete oncological response. A 50% decline of anti-HBs titers occurred in 22/63 (35%) patients, including 12 who became anti-HBs seronegative. Five (6%) patients had ALT increase during R-CT but none required R-CT discontinuation. Seventeen (20%) patients died, all for tumour progression.

Conclusion: LMV prophylaxis is safe and effective in preventing HBV reactivation in HBsAg negative/anti-HBc positive NHL patients receiving R-CT.
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http://dx.doi.org/10.1016/j.dld.2018.08.024DOI Listing
March 2019

Global methylation patterns in primary plasma cell leukemia.

Leuk Res 2018 10 18;73:95-102. Epub 2018 Sep 18.

Unit of Hematology and Stem Cell Transplantation, IRCCS-CROB, Referral Cancer Center of Basilicata, Rionero in Vulture, Italy.

Primary plasma cell leukemia (pPCL) is a rare and very aggressive variant of multiple myeloma (MM). Specific clinical, biological and molecular patterns distinguish pPCL from MM. Here, we performed a genome-wide methylation analysis by high-density array in 14 newly diagnosed pPCL patients along with 60 MMs, and 5 patients affected by monoclonal gammopathy of uncertain significance (MGUS). Our analysis revealed a global hypomethylation profile associated with pPCL. Additionally, differential methylation patterns were found related to distinct chromosomal aberrations and DIS3 mutations, affecting genes with roles in bone metabolism, cell migration, transcription regulation or DNA damage response. When compared with MM patients, pPCL showed a distinct methylation profile mostly characterized by hypomethylated probes specific for genes involved in several processes like cell adhesion and migration. Furthermore, decreasing methylation levels were evidenced for genes significantly modulated in the progressive phases of plasma cell dyscrasias, from MGUS to MM and pPCL. Overall, our data provide new insights into the molecular characterization of pPCL, thus being potentially useful in the prognostic stratification or identification of novel molecular targets.
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http://dx.doi.org/10.1016/j.leukres.2018.09.007DOI Listing
October 2018

Evaluation of the Impact of Cognitive Training on Quality of Life in Patients with Multiple Sclerosis.

Eur Neurol 2017 24;78(1-2):111-117. Epub 2017 Jul 24.

Objective: The aim of this study was to evaluate the effectiveness of cognitive rehabilitation in a group of multiple sclerosis (MS) patients.

Methods: Thirty-four patients were included in this study and randomly allocated either to treatment with multidisciplinary rehabilitation plus cognitive training or to treatment with multidisciplinary rehabilitation alone.

Results: After 3 months of cognitive treatment, the patients assigned to the rehabilitation plus cognitive training group displayed an improvement in the cognitive test of executive function and a marked improvement in quality of life (QoL). The patients treated with multidisciplinary rehabilitation without cognitive training improved in the physical composite score alone. Both groups of patients displayed an improvement in depression, though the improvement was confirmed at the 6-month follow-up examination (p = 0.036) only in patients treated with multidisciplinary rehabilitation plus cognitive training.

Conclusions: Our results indicate that the multidisciplinary rehabilitation treatment is the best approach to treat MS. The specific effect of each treatment needs to be assessed to be able to determine its role within a multidisciplinary approach. Cognitive rehabilitation is an important aspect of this multidisciplinary approach insofar as it may improve the QoL of MS people.
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http://dx.doi.org/10.1159/000478726DOI Listing
April 2018

Primary plasma cell leukemia 2.0: advances in biology and clinical management.

Expert Rev Hematol 2016 Nov 24;9(11):1063-1073. Epub 2016 Oct 24.

e Scientific Direction , IRCCS-CROB, Referral Cancer Center of Basilicata , Rionero in Vulture , Italy.

Introduction: Primary plasma cell leukemia (PPCL) is a rare and aggressive variant of multiple myeloma. The introduction of novel agents and modern technologies has recently partially changed the clinical and biological scenario of this malignancy, allowing limited, but not negligible, progresses. Areas covered: We will discuss: the complex landscape of genetic alterations in PPCL, derived from conventional and high-throughput technologies; the best available treatments for PPCL; the possible future therapeutic perspectives. Expert commentary: PPCL requires an immediate and intensive multi-phase treatment with short therapy-free intervals, which should include novel agents and autologous stem cell transplantation in eligible patients. Allogeneic transplantation should be considered in selected cases. In older and/or frailer individuals, personalized approaches should be applied. Integrated treatments with next generation proteasome inhibitors/IMIDs and monoclonal antibodies are currently planned or under investigation. The identification of novel genomic biomarkers may be potentially helpful for risk stratification and future personalized therapies.
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http://dx.doi.org/10.1080/17474086.2016.1244002DOI Listing
November 2016

Does robot-assisted gait training improve ambulation in highly disabled multiple sclerosis people? A pilot randomized control trial.

Mult Scler 2017 Apr 11;23(5):696-703. Epub 2016 Aug 11.

Multiple Sclerosis Unit, IRCCS Santa Lucia Foundation, Rome, Italy.

Background: Robotic training is commonly used to assist walking training in patients affected by multiple sclerosis (MS) with non-conclusive results.

Objective: To compare the effect of robot-assisted gait training (RAGT) with that of conventional walking training (CWT) on gait competencies, global ability, fatigue and spasticity in a group of severely affected patients with MS.

Methods: A pilot, single-blind randomized controlled trial was conducted in 43 severe (Expanded Disability Status Scale (EDSS) score of 6-7.5) and non-autonomous ambulant in-patients with MS. Experimental group performed 12 sessions of RAGT, whereas control group performed the same amount of CWT. Primary outcome measures were gait ability assessed by 2 minutes walking test and Functional Ambulatory Category; secondary outcomes were global ability (modified Barthel Index), global mobility (Rivermead Mobility Index), severity of disease (EDSS) and subjectively perceived fatigue (Fatigue Severity Scale).

Results: The number of subjects who achieved a clinical significant improvement was significantly higher in RAGT than in CWT ( p < 0.05 for both primary outcome measures). RAGT also led to an improvement in all the other clinical parameters (global ability: p < 0.001, global mobility: p < 0.001, EDSS: p = 0.014 and fatigue: p = 0.001).

Conclusions: RAGT improved the walking competencies in non-autonomous ambulant patients with MS, with benefits in terms of perceived fatigue.
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http://dx.doi.org/10.1177/1352458516663033DOI Listing
April 2017

Chronic Pain in Multiple Sclerosis Patients: Utility of Sensory Quantitative Testing in Patients with Fibromyalgia Comorbidity.

Eur Neurol 2015 2;73(5-6):257-63. Epub 2015 Apr 2.

IRCCS Fondazione 'Santa Lucia'; UO 'E' Multiple Sclerosis Unit, Rome, Italy.

Background: Lower thermal and discomfort thresholds may predispose multiple sclerosis (MS) patients to chronic pain, but a possible effect of fibromyalgia (FM) comorbidity has never been investigated. Aims were to investigate the thermal and discomfort thresholds in the evaluation of pain intensity between MS patients with FM (PFM+) and MS patients with pain not associated to FM (PFM-).

Methods: One hundred thirty three MS patients were investigated for chronic pain. FM was assessed according to the 1990 ACR diagnostic criteria. An algometer was used to measure the thresholds in the patients and 60 matched healthy subjects.

Results: Chronic pain was present in 88 (66.2%) patients; 12 (13.6%) had neuropathic pain, 22 (17.3%) were PFM+ and 65 (48.9%) PFM-. PFM+ were predominantly female (p = 0.03) and had a greater EDSS (p = 0.01) than NoP; no other significant differences emerged than PFM-. The thresholds were lower in MS patients than controls (p < 0.01), mainly in the PFM+. FM severity influenced the thermal threshold (p < 0.001), while the female gender influenced the discomfort threshold (p < 0.001).

Conclusion: Thermal and discomfort thresholds were lower in patients than controls and were the lowest in PFM+. Their more severely impaired thermal threshold supports a neurophysiological basis of such association.
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http://dx.doi.org/10.1159/000381211DOI Listing
January 2016

Chronic pain in multiple sclerosis: is there also fibromyalgia? An observational study.

Med Sci Monit 2014 May 9;20:758-66. Epub 2014 May 9.

Multiple Sclerosis Unit, I.R.C.C.S. Fondazione "Santa Lucia", Rome, Italy.

Background: Chronic pain is common in persons with multiple sclerosis (MS), but the co-morbidity of fibromyalgia (FM) has yet to be investigated in MS. Objectives of the study were to evaluate, among the various types of chronic pain, the frequency of FM in MS and its impact on MS patients' health-related quality of life (HRQoL).

Material And Methods: 133 MS patients were investigated for the presence and characterization of chronic pain within 1 month of assessment. A rheumatologist assessed the presence FM according to the 1990 ACR diagnostic criteria. Depression, fatigue, and HRQoL were also assessed by means of specific scales.

Results: Chronic pain was present in 66.2% of patients (musculoskeletal in 86.3%; neuropathic in 13.7%; absent in 33.8% [called NoP]). Pain was diagnosed with FM (PFM+) in 17.3% of our MS patients, while 48.9% of them had chronic pain not FM type (PFM-); the prevalence of neuropathic pain in these 2 sub-groups was the same. PFM+ patients were prevalently females and had a higher EDSS than NoP. The PFM+ patients had a more pronounced depression than in the NoP group, and scored the worst in both physical and mental QoL.

Conclusions: In our sample of MS patients we found a high prevalence of chronic pain, with those patients displaying a higher disability and a more severe depression. Moreover, FM frequency, significantly higher than that observed in the general population, was detected among the MS patients with chronic pain. FM occurrence was associated with a stronger impact on patients' QoL.
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http://dx.doi.org/10.12659/MSM.890009DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4026145PMC
May 2014

Psychological care of caregivers, nurses and physicians: a study of a new approach.

Cancer Med 2014 Feb 4;3(1):101-10. Epub 2014 Jan 4.

Division of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.

There is much evidence demonstrating that psychosocial interventions in caregivers and oncological staff produce an improvement in their patients' quality of life. The aim of this explorative study was to evaluate the effect of a new approach in promoting more functional ways to face stressful situations in the constellation of people around patients: caregivers, physicians and nurses. Thirty-four subjects were divided into three groups: 10 caregivers, 11 physicians, and 13 nurses. A "Balint Group" method modified according to a mindfulness technique was used as the intervention. Three assessment tools were administered to the participants at baseline, during, and after completion of the study: the Response Evaluation Measure (REM-71), the Satisfaction Profile (SAT-P), and the Group Climate Questionnaire (GCQ). Mean values of defense mechanisms determined by the REM-71 were compared with those of the standard population. At baseline, we observed a prevalence of immature defenses in the three groups, with mean values above those in the standard population. After the psychological intervention, a tendency to normalization of the mean values was observed, indicating the development of more adaptive ways of using defense mechanisms and the effectiveness of the intervention. Group climate, assessed through the GCQ, showed an increase in the "Engagement" factor and a decline in the "Conflict" factor in all groups. This study suggests that group treatment focused on changing personal responses to stressful situations can induce more adaptive strategies enabling caregivers, hematologists, and nurses to help patients better and thereby improve their quality of life.
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http://dx.doi.org/10.1002/cam4.163DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3930394PMC
February 2014

Long term evaluation of the impact of autologous peripheral blood stem cell transplantation in multiple myeloma: a cost-effectiveness analysis.

PLoS One 2013 30;8(9):e75047. Epub 2013 Sep 30.

Division of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.

Background: High-dose therapy with autologous peripheral stem cell transplantation represents today the standard approach for younger multiple myeloma patients. This study aimed to evaluate the long term economic impact of autologous transplantation with respect to conventional therapy.

Methods: We retrospectively reviewed the charts of multiple myeloma patients diagnosed at our department between 1986 and 2003 and treated according to the therapy considered standard at the time of diagnosis. Analysis of costs was done by assessing resource utilization and direct costs were measured and monetized before proceeding with the analysis, based on public health service tariffs.

Results: Group A including 78 patients treated with Melphalan and Prednisone was compared with Group B including 74 patients who received an autologous transplant. The median overall survival was 3.2 and 5.4 years respectively (p = 0.0002). Mean cost per patient was significantly higher in group B with respect to group A (102373€ vs 23825€; p<0.001). The final quality-adjusted-life-year gain in group B patients as compared to group A was 1.73 QALY, with an incremental cost-effectiveness ratio of 45460€. With a threshold of 75000€ per QALY gained, the cost effectiveness acceptability curve indicated that the probability that autologous transplantation in multiple myeloma is a cost-effective intervention is 90%.

Conclusions: The cost of autologous transplantation remains high. The calculated incremental cost-effectiveness ratio, however, given the significant prolongation of overall survival obtained with autologous transplantation, is within an acceptable threshold. Notwithstanding, its high cost should be taken into account when considering the whole cost of multiple myeloma.
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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0075047PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3787096PMC
May 2014

Monoclonal gammopathy of undetermined significance: a new proposal of workup.

Eur J Haematol 2013 Oct 17;91(4):356-60. Epub 2013 Aug 17.

Division of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.

Objective: Diagnostic criteria for monoclonal gammopathy of undetermined significance (MGUS) require quantification of bone marrow plasma cells (BMPCs) and skeletal survey to discriminate between MGUS and multiple myeloma (MM). By contrast, recent published guidelines suggest that these procedures could be avoided in the presence of serum monoclonal spike (M-spike) of small amount (≤1.5 g/dL). Aim of this study is to better quantify the risk of missing a diagnosis of MM, not performing bone marrow aspirate and skeletal survey in patients with M-spike ≤ 1.5 g/dL asymptomatic for bone pain.

Methods: We reviewed data of 2282 patients consecutively observed from January 1974 to December 2010 in our single hematology department. We considered eligible for this study 1271 patients with grade <2 NCI bone pain, confirmed to have an MGUS or an MM after extensive standardized diagnostic workup including bone marrow biopsy, skeletal bone survey and laboratory tests.

Results: The risk of finding a BMPC infiltration ≥10% in patients with an M-spike ≤ 1.5 g/dL was very low (7.3%), although significantly different according to IgH isotype (4.7% for IgG vs. 20.5% for IgA). The risk of finding bone lesions with M-spike ≤ 1.5 g/dL was negligible (2.5%), regardless of IgH isotype.

Conclusion: In asymptomatic patients with M-spike of small amount (≤1.5 g/dL): (i) BMPC evaluation may be reasonably avoided in patients with IgG M-spike, while should always be part of diagnostic workup in the presence of IgA M-spike and (ii) skeletal survey, less predictive for MM, should not be routinely indicated irrespective of IgH isotype.
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http://dx.doi.org/10.1111/ejh.12172DOI Listing
October 2013

Good clinical activity and favorable toxicity profile of once weekly bortezomib, fotemustine, and dexamethasone (B-MuD) for the treatment of relapsed multiple myeloma.

Am J Hematol 2013 Feb 8;88(2):102-6. Epub 2012 Dec 8.

Division of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy.

Since multiple myeloma (MM) is still not-curable, the management of relapse remains challenging. Given the known efficacy of alkylating agents in MM, we conducted a phase I/II study to test a new three drug combination in which Fotemustine (Muphoran), an alkylating agent of nitrosurea family, was added to bortezomib + dexamethasone backbone (B-MuD) for the treatment of MM relapsed patients. Fotemustine was administered at two dose levels (80-100 mg/m² i.v.) on day 1. The original 21-day schedule was early amended for extra-hematological toxicity and a 35-day schedule was adopted (Bortezomib 1.3 mg/m² i.v. on days 1, 8, 15, and 22, Dexamethasone 20 mg i.v. on days 1, 8, 15, and 22) for a total of six courses. Twenty-four patients were enrolled. The maximum tolerated dose of Fotemustine was 100 mg/m². The overall response rate was of 62% (CR 8%, VGPR 33%, and PR 21%). The median OS was 28.5 months, the median progression-free survival (PFS) was 19.1 months. B-MuD resulted effective in patients previous exposed to bortezomib without difference of response (P = 0.25) and PFS (P = 0.87) when compared to bortezomib-naive patients. Thrombocytopenia was the most common AE overall. In conclusion, B-MuD is an effective and well tolerated combination in relapsed MM patients even in advanced disease phase.
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http://dx.doi.org/10.1002/ajh.23358DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3563219PMC
February 2013

Survey of current practice for monitoring and management of platelet refractoriness in Italy.

Transfus Apher Sci 2012 Dec 28;47(3):271-6. Epub 2012 May 28.

Department of Transfusion Medicine, Pescara Civil Hospital, Pescara, Italy.

Platelet transfusion failure is a common phenomenon affecting from 7% to 34% of haematology-oncology patients. Monitoring the efficacy of platelet transfusion through the evaluation of a post-transfusion platelet count and clinical response represent an important guide for subsequent transfusions and for the detection of refractoriness. The aim of this survey was to investigate physicians' attitudes and practices regarding the monitoring of platelet response and the management of platelet refractoriness. An e-mail based survey was conducted among the heads of blood banks with a hemapheresis ward in Italy. Heads of 64 centers out of the 122 initially identified (52%) completed the entire survey. Apheresis, buffy-coat pool, and platelet rich plasma represented an average of 46%, 38% and 17% of the total number of transfusions, respectively. In the prophylaxis of hemorrhagic episodes, most of the centers utilized as standard dose one unit of apheresis platelets (55.7%) and/or one unit of buffy-coat pool platelets (42.6%), while 11.4% of respondents used an average of 6 units of platelet rich plasma. In only 27.9% of the centers was the platelet dose established based on the body weight of the recipient. Only one-third of the centers evaluated the response to platelet transfusion in all patients, while the rate increased to 60% in onco-hematological patients. Among patients transfused on an outpatient basis, the rate dropped to 20%, and a platelet sample taken 10 min after transfusion was generally used. The survey documented a substantial lack of interaction between the clinician requesting the transfusion and the one responsible for the preparation and delivery of the product, with both figures involved in the diagnosis of refractoriness in only one-third of the centers. In conclusion, despite being a frequent condition, platelet refractoriness is still managed with a high degree of heterogeneity and often overlooked. Better adherence to existing guidelines and standard operating procedures, as well as the involvement of transfusion centers in prospective evaluations can help reduce this variability and improve the outcome of transfused patients.
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http://dx.doi.org/10.1016/j.transci.2012.04.003DOI Listing
December 2012

Enhanced-MRI and ultrasound evaluation of painful shoulder in patients after stroke: a pilot study.

Eur Neurol 2011 3;66(3):175-81. Epub 2011 Sep 3.

Santa Lucia Foundation, IRCCS, Rome, Italy.

Background: Epidemiological and radiological studies have previously been performed to identify the possible causes of hemiplegic shoulder pain (HSP). Many different etiologies have been postulated, though no clear correlations have emerged, and a multifactorial pathogenesis of HSP has been proposed. Recently, two MRI-based studies have described different shoulder findings as possible causes of pain in chronic stroke survivors.

Purpose: The aim of this study was to describe the structural abnormalities of the painful shoulder in the first months after stroke by ultrasound and enhanced MRI. The secondary aims were to identify possible predisposing factors for HSP and to evaluate its impact on motor recovery.

Methods: One hundred and fifty-three first-time stroke patients, admitted to the Santa Lucia Foundation for rehabilitation, were investigated for HSP. Twenty-five stroke patients with HSP and 16 stroke patients without shoulder pain were included. An ultrasound evaluation and enhanced shoulder MRI were performed for all the patients.

Results: Among the shoulder abnormalities detected by both imaging studies, only capsulitis, which was detected by enhanced shoulder MRI in 88% of the HSP patients, was independently associated with pain (p < 0.001) and proven to be predictive of pain intensity as expressed by the VAS score (p < 0.003). HSP correlated with a worse global recovery (p < 0.05) as well as with male sex (p = 0.006), neglect (p = 0.02) and subluxation (p = 0.03), although none of these features were found to be independent predictors of pain.

Conclusion: Adhesive capsulitis was found to be a possible cause of HSP. However, MRI, which is more expensive than other diagnostic tools, may be considered the gold standard tool for understanding the etiology of HSP.
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http://dx.doi.org/10.1159/000330657DOI Listing
January 2012

A case of PANDAS treated with tetrabenazine and tonsillectomy.

J Child Neurol 2010 May 5;25(5):614-5. Epub 2010 Mar 5.

Santa Lucia Foundation IRCCS Hospital, Department F and Laboratory of Neuroanatomy, Santa Lucia Foundation IRCCS at the European Center for Brain Research, Rome, Italy.

PANDAS (pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections) is a rare clinical syndrome characterized by the presence of tics, Tourette syndrome, obsessive-compulsive disorder, or chorea in the context of an immediately precedent streptococcal infection. In this report, we describe the case of an 11-year-old boy who developed PANDAS with severe choreic movements. The criteria for PANDAS diagnosis were met. Moreover, serum antibrain antibodies were present. The patient was initially treated with tetrabenazine 12.5 mg twice daily with remission of the neurological symptoms. Subsequently, the patient underwent tonsillectomy and has been asymptomatic since, with antistreptolysin O titer levels in range.
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http://dx.doi.org/10.1177/0883073809355824DOI Listing
May 2010