Kooperativer Bibliotheksverbund

In: eJHaem, Wiley, Vol. 1, No. 1 ( 2020-07), p. 384-387

Type of Medium: Online Resource

ISSN: 2688-6146 , 2688-6146

URL: Issue

URL: Article

DOI: 10.1002/jha2.v1.1

DOI: 10.1002/jha2.53

Language: English

Publisher: Wiley

Publication Date: 2020

detail.hit.zdb_id: 3021452-X

In: Blood, American Society of Hematology, Vol. 114, No. 22 ( 2009-11-20), p. 3717-3717

Abstract: Abstract 3717 Poster Board III-653 Introduction Primary cutaneous lymphomas (PCL) present in the majority of histologic variants an indolent behaviour and a good prognosis with a prolonged survival. In a very small number of patients (pts), PCL is aggressive at the onset while in pts who are resistant or have relapsed after repeated topic or systemic therapies an advanced disease is more frequently observed. In these patients no therapy is capable of inducing a stable remission of the disease. The efficacy and low toxicity of Pegylated Liposomal Doxorubicin (PLD) as a single agent in second-line therapy of PCL was recently demonstrated. In our study we tested the safety and efficacy of PLD (Caelyx®) (C) in association with three drugs of proven effectiveness in nodal lymphoprolipherative and other primary cutaneous neoplastic disorders: Bleomycin (B), Vinblastine (B) and Dacarbazine (D) (CBVD). Patients and Methods From February 2003 to December 2008 we observed 37 consecutive pts with advanced PCL: 19 Cutaneous T-Cell Lymphomas (CTCL) and 18 cutaneous B-Cell Lymphomas (CBCL). The CTCL pts were: 15 males and 4 females, with median age 59 (27-86) years, of which 7 pts with Anaplastic Large Cell Lymphoma (ALCL) CD30 positive, 7 pts with transformed Mycosis Fungoides (tMF), 3 pts with CD30 negative ALCL, 1pt with Panniculitis-like (Pl) Lymphoma and 1 pt with transformed Sezary Syndrome (tSS), according to the WHO-EORTC consensus classification. Nine pts presented with a nodal involvement and 10 pts were resistant or relapsed after 1 or more systemic treatments. Among the CBCL pts there were: 8 males and 10 females, with median age 61 (42-84) years, 12 pts had a Follicular Centre Lymphoma (FCL), 3 a Marginal Zone Lymphoma (MZL) and 3 a Diffuse Large B-Cell Lymphoma Leg Type (LT). Five pts presented a nodal involvement and 11 pts had received 1 or more previous systemic therapy. All 37 pts received CBVD therapy at following dosage: C: 20 mg/m2, B: 10 mg/m2, V: 6 mg/m2, D: 325 mg/m2 at days 1 and 15, administered intravenously every 4 weeks for a maximum of 6 cycles. Rituximab (R) at dosage of 375 mg/m2 was administered to CBCL pts at 1st day of each cycle. Before the treatment, pts were submitted to a complete staging of disease including TC-scan, bone marrow biopsy, and immunophenotyping of peripheral blood cells. Results In the CTCL group 4 pts received 4 CBVD cycles, 14 pts received 6 CBVD cycles and 1 pt presented a progressive disease after the 1st cycle. Overall Response Rate (ORR) was 94.7% (18/19 pts). Sixteen out of 18 pts (88.8%) had a Complete Remission (CR) with disappearance of nodal involvement and cutaneous lesions, 2/18 pts obtained a Partial Remission (PR) with disappearance of nodal involvement and 75% of cutaneous lesions. In the CBCL group 3 pts received 2 R-CBVD cycles, 3 pts 4 cycles and 11 pts 6 cycles: ORR was 100% and all pts obtained a CR. The occurrence of palmoplantar erytrodisesthesia in 5 pts and grade 2-3 granulocytopenia in 12 pts did not modify the therapy program. Two CTCL pts (1 tSS, 1Pl) and 1 CBCL (LT) pt in CR after therapy received an allogeneic Hemopoietic Stem Cells (HCS) transplant and 1 CBCL(FCL) pt a syngeneic HCS transplant. In CTCL group 11 (61%) pts maintained their response after a median follow up of 15.5 (9-71) months. Among CBCL pts 14 (77.7%) pts are still in CR after a median follow up of 15 (4-36) months. Conclusions Our experience demonstrates that the CBVD association is an effective and safe therapy for advanced PCL in inducing an important tumour burden reduction with a high CR rate. The assessment of response duration requires a longer observation. A larger number of patients in a multicentric trial are needed to confirm our promising results. Disclosures: Off Label Use: liposomial doxorubicin has a peculiar cutaneous tropism and low cardiac toxicity compared with other anthracyclines.

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood.V114.22.3717.3717

Language: English

Publisher: American Society of Hematology

Publication Date: 2009

detail.hit.zdb_id: 1468538-3

detail.hit.zdb_id: 80069-7

In: Blood, American Society of Hematology, Vol. 118, No. 21 ( 2011-11-18), p. 2651-2651

Abstract: Abstract 2651 Background and objectives [18F]fluorodeoxyglucose - Positron Emission Tomography (FDG-PET) is confirmed as a useful functional imaging tool for staging and response assessment in Hodgkin lymphoma and Diffuse Large B-Cell Lymphoma. Despite FL is accounted among FDG-avid lymphomas few studies have been performed to investigate how FDG-PET can be used in initial staging of FL patients. We conducted a retrospective analysis to investigate the role of FDG-PET in the initial staging of FL. Patients and methods The study was designed as a retrospective unplanned analysis of patients with newly diagnosed FL enrolled in the FOLL05 phase III trial (NCT00774826) and randomized to one of the three study arms (R-CVP. R-CHOP, R-FM). To be included in the study patients should have confirmed eligibility for the FOLL05 trial, have available data on clinical presentation, treatment details and results, and on follow-up. Baseline staging had to be performed with contrast-enhanced Computed Tomography (CT), with CT-PET, and with Bone Marrow (BM) biopsy. For study purposes disease extension at baseline was defined independently for both CT and PET using on local report and interpretation; only for difficult cases images were centralized and reviewed. For each exam nodal sites (NS) were counted according to the FLIPI schema. NS were considered as positive if greater than 1.5 cm in their maximum transverse diameter at CT or, using PET, if FDG avid or if they had disappeared at the end of treatment. Extranodal sites (ENS) were counted on an organ basis and were considered positive at CT in case of nodular involvement or in case of organ enlargement not otherwise justified. Extranodal involvement at PET was considered for sites showing avidity for FDG not justified by conditions other than FL. Conventional Ann Arbor (AA) staging was based on CT scan assessment only. PET and CT scan results were compared using the kappa-statistic measure of interrater agreement (IR), and the level of agreement was defined by the Koch Landis scale. Results Among 534 patients enrolled in the FOLL05 trial, 122 cases fulfilled eligibility criteria for this study. All but 2 cases were confirmed as FDG avid at PET scan; these two cases were not used for staging comparison. Median age of patients was 57 years (range 33–74), 33% were older than 60 years, 48% were males. Bone marrow biopsy was positive in 52%. Using CT, AA stage was III-IV in 77% of cases. Fifty-two percent, 36%, and 12% of cases had less 0–4, 5–8, and 〉 8 NS, at CT, respectively. Overall CT scan allowed the identification of 48 ENS in 36 patients (30%); 2 or more ENS were described in 8 patients (6%); most frequent ENS were spleen (52%), liver (10%), skin/soft tissue (8%) and GI tract (6%). Using PET 38%, 37%, and 25% of cases had 0–4, 5–8, 〉 8 NS, respectively. PET allowed the identification of 88 ENS in 55 patients (46%) and 2 or more ENS were found in 17 patients (14%). Most frequent ENS at PET were bone (35%), spleen (30%), GI tract (10%), and skin/soft tissue (7%). Classifying patients according to the number of NS (0–4, 5–8, 〉 8) agreement between CT and PET was fair (IR= 61%, Kappa=0.39). Agreement between CT and PET for ENS (grouped as 0, 1, and 〉 1) was also fair (IR= 63%, Kappa=0.31) and improved to moderate when also details on BM histology were considered (IR 82% K=0.4). When PET was used independently of CT to define AA stage a moderate agreement was achieved with CT (IR=76%, kappa= 0.58): in particular 22 cases (18%) were upstaged with PET while only 6 (5%) were downstaged; seventeen out of 25 (68%) patients were reclassified by PET as stage III-IV from a previous localized stage. Looking at FLIPI, 22%, 41%, and 37% were classified by CT as having score of 0–1, 2, and 3–5, respectively. The use of PET modified CT based FLIPI index in 26% of cases, with a substantial agreement between PET and CT (IR=74%, kappa=0.61). FLIPI2 index is not affected by the use of PET. Conclusions The results of this study confirm FL as a FDG-avid disease. The use of PET for the initial staging of patients with FL seems to provide a more accurate definition of disease extent compared with CT. The clinical usefulness of adding PET to the initial staging of FL needs to be further investigated. Disclosures: Di Raimondo: Celgene: Honoraria, Speakers Bureau; Janssen: Honoraria, Speakers Bureau.

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood.V118.21.2651.2651

Language: English

Publisher: American Society of Hematology

Publication Date: 2011

detail.hit.zdb_id: 1468538-3

detail.hit.zdb_id: 80069-7

In: Hematological Oncology, Wiley, Vol. 33, No. 3 ( 2015-09), p. 147-151

Abstract: We conducted a population‐based study to establish the outcome of T‐cell lymphoma (TCL) patients failing systemic first‐line therapy. All TCL patients failing first‐line systemic therapy in the province of Modena were identified from Modena Cancer Registry between 1997 and 2010. A total of 53 patients were analysed. Regarding the type of failure, 18 patients relapsed, and 35 progressed during first treatment. Among relapsed patients, the median time from date of response to relapse after first treatment was 6.2 months (range 1.87–102). A total of 18 patients (34%) died before receiving salvage treatment, 21 received platinum or gemcitabine‐containing regimens (7 addressed to autologous stem cell transplant (ASCT)), 12 other CT regimens; 2 received radiotherapy (RT). The median survival after relapse (SAR) was 2.5 months. After a median follow‐up for living patients after failure of 35 months (range 8–111 months), 44 patients died, and the cause of death was found to be lymphoma progression in all (98%) but one of them. The median SAR was 2.5 months. The 3‐year SAR was 19%. Univariate and multivariate Cox regression analyses for SAR were performed. In multivariate analysis, performance status and type of failure were associated with a higher risk of death after relapse. The outcome of TCL patients failing first‐line therapy is poor. Only a few cases that could receive ASCT had promising chances of long remission. There is urgent need for novel agents for patients requiring second‐line treatment. Copyright © 2014 John Wiley & Sons, Ltd.

Type of Medium: Online Resource

ISSN: 0278-0232 , 1099-1069

URL: Issue

URL: Article

DOI: 10.1002/hon.v33.3

DOI: 10.1002/hon.2144

Language: English

Publisher: Wiley

Publication Date: 2015

detail.hit.zdb_id: 2001443-0

In: Hematological Oncology, Wiley, Vol. 39, No. 1 ( 2021-02), p. 41-50

Abstract: Carfilzomib–lenalidomide–dexamethasone (KRd) has been approved for the treatment of relapsed/refractory multiple myeloma (RRMM). We conducted a retrospective analysis of 197 RRMM patients (pts) between January 2016 and March 2018 in six Italian hematologic centers, with the aim to evaluate efficacy and safety of KRd in real‐life. At KRd initiation 27% carried high risk cytogenetic abnormalities (HRCA) [del17p and/or t(4;14) and/or t(14;16)], median number of prior lines of therapy was 2 (1–8), nearly all pts (96%) received prior bortezomib (18% refractory) while 45% were exposed to lenalidomide (R; 22% refractory). At the median of 12.5 months, 52% of the pts had discontinued treatment, mainly (66%) for progression. Main grade 3–4 adverse events were neutropenia (21%), infections (11%), and hypertension (6%). Overall, the response rate was 88%. The median progression‐free survival (PFS) was 19.8 months and 1‐year overall survival (OS) rate was 80.6%. By subgroup analysis, extended PFS and OS were observed for pts who received ≤2 prior lines of therapy (HR = 0.42, p 〈 0.001 and HR = 0.35, p = 0.001, respectively), not refractory to prior R (HR = 0.37, p 〈 0.001, and HR = 0.47, p = 0.024), without HRCA (HR = 0.33, p = 0.005 and HR = 0.26, p = 0.016) and achieving ≥ very good partial response (VGPR; HR = 0.17, p 〈 0.001 and HR = 0.18, p 〈 0.001). In conclusion, KRd demonstrated to be effective in RRMM pts treated in real‐world setting, without new safety concerns. Better survival outcomes emerged for pts with ≤2 prior lines of therapy, achieving at least a VGPR, and without HRCA.

Type of Medium: Online Resource

ISSN: 0278-0232 , 1099-1069

URL: Issue

URL: Article

DOI: 10.1002/hon.v39.1

DOI: 10.1002/hon.2820

Language: English

Publisher: Wiley

Publication Date: 2021

detail.hit.zdb_id: 2001443-0

In: British Journal of Haematology, Wiley, Vol. 163, No. 1 ( 2013-10), p. 40-46

Abstract: Novel treatments for multiple myeloma ( MM ) have shown promising results in clinical trials, but the advantage in unselected patients is still unclear. In order to evaluate whether novel therapies impact survival of MM patients, we performed a population‐based analysis on data collected by the M odena C ancer R egistry from 1989 to 2009. The analysis evaluated 1206 newly diagnosed MM patients collected in the years 1988–96 (conventional therapy), 1997–05 (high dose melphalan and autologous transplant), and 2006–09 (novel agents era). Both relative survival ( RS ) and overall survival ( OS ) improved over the years, but not equally in the three groups. For patients aged 〈 65 years, RS improved in 1997–05 and 2006–09 compared with previous years and a trend to improvement was observed from 1997–05 to 2006–09. For patients aged 65–74 years, RS improved significantly in 2006–09 compared with 1988–96 and 1997–05. No amelioration was observed for patients 75+ years old. OS confirmed RS . In conclusion, the survival of MM patients aged 〈 65 and, in particular, 65–74 years, has improved over time, especially after 2006. This observation provides circumstantial evidence that novel therapies might impact patient survival. Despite the limits of this study, these data refer to an unselected population, giving a picture of every day clinical practice.

Type of Medium: Online Resource

ISSN: 0007-1048 , 1365-2141

URL: Issue

URL: Article

DOI: 10.1111/bjh.2013.163.issue-1

DOI: 10.1111/bjh.12465

Language: English

Publisher: Wiley

Publication Date: 2013

detail.hit.zdb_id: 1475751-5

In: Blood, American Society of Hematology, Vol. 112, No. 11 ( 2008-11-16), p. 1519-1519

Abstract: Implementation of transplant procedures and the availability of alternative sources of stem cells for patients without HLA identical siblings has made allogeneic stem cell transplant (ASCT) a suitable therapy for many patient affected by acute myeloid leukemia (AML); consequently, in AML patients transplant strategies must be carefully designed according to stringent risk/benefit analysis. To date, risk assessment relies on identification of baseline prognostic parameters such as karyotype, which has become critical in order to choose post-remissional therapy. Furthermore, there are strong evidences that the presence of specific gene abnormalities, such as mutations of FLT3, NPM and c-kit, allow to identify, within homogeneous cytogenetic groups, subsets of patients with distinct clinical outcome. We hypothesized that, in order to guide the post-remission decisional process, more robust informations may derive from the combined evaluation of conventional baseline and delayed prognosticators. Minimal residual disease (MRD) detection is potentially the most efficient tool to investigate the quality of remission and might represent the ideal parameter to be associated with baseline biological features for prognostic purposes. Therefore, we aimed to determine whether combined analysis of karyotype and MRD, using multiparametric flow-cytometry (MPFC), could help to refine risk assessment in adult patients with AML, allowing the most appropriate post-remissional strategy to be selected. We analyzed 132 patients with AML who had intensive chemotherapy with EORTC/GIMEMA protocols. According to MRC classification, 22 (17%), 104 (78%) and 6 (5%), respectively, had good, intermediate and poor risk karyotype. Thirteen of 107 (12%) carried a FLT3-ITD. Within good and intermediate risk categories, MRD positivity (≥ 3.5×10−4 residual leukemic cells at the post-consolidation time-point) was associated with a worse prognosis in terms relapse free (RFS) and overall survival (OS). In fact, we observed that: MRD negative good and intermediate risk categories shared the same favorable prognoses with RFS and OS of 80% vs. 69% and 84% vs 54%, respectively; MRD positive good and intermediate risk categories fared as worse as those with poorrisk karyotype or FLT3-ITD in terms of RFS (28% vs. 17% vs. 0%) and OS (42% vs. 21% vs. 17%). Using this approach the conventional cytogenetic classification that uses three categories is simplified into 2 prognostically defined groups: favorable, including MRD negative good and intermediate risk karyotype; unfavorable, including MRD positive good and intermediate risk karyotype, poor risk karyotype and FLT3-ITD positive cases (Fig. 1). Based on these observations, we believe that ASCT is recommended, not only for poor-risk karyotype or FLT3 positive AML, but also for good/intermediate risk categories not gaining MRD negativity, being this option able to provide a superior chance of prolonged RFS (Maurillo et al, JCO 2008). On the other hand, patients belonging to MRD negative good/intermediate risk categories, who can experience a 5-years survival higher than 60%, may have their life expectancy hampered by the choice of a therapeutic strategy with a disadvantageous risk/benefit ratio: for this category a standard intensification procedure (chemo and/or autologous transplant) is indicated. In conclusion, the combined assessment of baseline prognosticators (cytogenetics) and parameters inherent the quality of response (MRD), is useful to define discrete categories of risk within the respective karyotypic groups, allowing tailored therapeutic approaches to be applied according to the actual clinical risk and avoiding under or over treatments. Figure 1 Figure 1.

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood.V112.11.1519.1519

Language: English

Publisher: American Society of Hematology

Publication Date: 2008

detail.hit.zdb_id: 1468538-3

detail.hit.zdb_id: 80069-7

In: Blood, American Society of Hematology, Vol. 136, No. Supplement 1 ( 2020-11-5), p. 35-37

Abstract: Background. Proteasome inhibitor (PI)-based induction/consolidation proved to be effective in newly diagnosed multiple myeloma (NDMM) patients (pts) eligible for melphalan 200 mg/m2 plus autologous stem-cell transplantation (MEL200-ASCT). High response rates have been reported with carfilzomib (K) plus lenalidomide-dexamethasone (KRd) or cyclophosphamide-dexamethasone (KCd). Lenalidomide (R) alone is a standard of care for post-ASCT maintenance; K maintenance showed promising results in phase I/II studies, but no data on KR maintenance vs R are available. Aims. The aims of this analysis were to evaluate the progression-free survival (PFS) of KRd induction-ASCT-KRd consolidation (KRd_ASCT) vs 12 cycles of KRd (KRd12) vs KCd induction-ASCT-KCd consolidation (KCd_ASCT) and the PFS of KR vs R maintenance. Secondary aims were efficacy in different subgroups of pts and safety of the maintenance phase. Methods. NDMM pts ≤65 years were randomized [R1: 1:1:1, stratification International Staging System (ISS) and age] to: KRd_ASCT: 4 28-day cycles with KRd induction (K 20/36 mg/m2 IV days 1,2,8,9,15,16; R 25 mg days 1-21; dexamethasone [d] 20 mg days 1,2,8,9,15,16) followed by MEL200-ASCT and 4 KRd consolidation cycles; KRd12: 12 KRd cycles; KCd_ASCT: 4 28-day induction cycles with KCd (K 20/36 mg/m2 IV days 1,2,8,9,15,16; cyclophosphamide 300 mg/m2 days 1,8,15; d 20 mg days 1,2,8,9,15,16) followed by MEL200-ASCT and 4 KCd consolidation cycles. Thereafter, pts were randomized (R2) to maintenance with KR (K 36 mg/m2 days 1,2,15,16, subsequently amended to 70 mg/m2 days 1,15 for up to 2 years; plus R 10 mg days 1-21 every 28 days until progression) or R alone (10 mg days 1-21 every 28 days until progression). Centralized minimal residual disease (MRD) evaluation (8-color second-generation flow cytometry, sensitivity 10-5) was performed in pts achieving ≥very good partial response before maintenance and every 6 months (m) during maintenance. Data cut-off was June 30, 2020. Results. 474 NDMM pts were randomized (KRd_ASCT, n=158; KRd12, n=157; KCd_ASCT, n=159) and analyzed. Pt characteristics were well balanced. Intention-to-treat (ITT) data of pre-maintenance MRD (KRd_ASCT, 62%; KRd12 56%, KCd_ASCT 43%) and safety of the induction/consolidation phases in the 3 arms were already reported (F. Gay et al. ASH 2018; S. Oliva et al. ASH 2019). After a median follow-up from R1 of 45 m, median PFS was not reached with KRd_ASCT, 57 m with KRd12 and 53 m with KCd_ASCT (KRd_ASCT vs KCd_ASCT: HR 0.53, P & lt;0.001; KRd_ASCT vs KRd12: HR 0.64, P=0.023; KRd12 vs KCd_ASCT: HR 0.82, P=0.262). The benefit of KRd_ASCT vs both KCd_ASCT and KRd12 was observed in most subgroups (Figure). 3-year overall survival (OS) was 90% with KRd_ASCT and KRd12 vs 83% with KCd. 356 pts (KR, n=178; R, n=178) were randomized to maintenance; pt characteristics, pre-maintenance response (≥complete response [CR]: KR 62% vs R 59%; stringent CR: KR 50% vs R 48%) including MRD negativity (KR 65% vs R 66%) in the 2 groups were well balanced. After a median follow-up from R2 of 31 m and a median duration of maintenance of 27 m in both arms, 46% of MRD-positive pts at randomization turned negative in KR vs 32% in R (P=0.04). By ITT analysis, 3-year PFS from R2 was 75% with KR vs 66% with R (HR 0.63; P=0.026). The benefit of KR vs R was observed in most subgroups (Figure). 3-year OS was 90% in both arms. During maintenance, a similar proportion of pts experienced ≥1 grade (G)3-4 hematologic adverse events (AEs)/serious AEs (SAEs) in the 2 arms (KR 22% vs R 23%); the most frequent were neutropenia (KR 18% vs R 21%) and thrombocytopenia (KR 3% vs R 3%). Rate of ≥1 G3-4 non-hematologic AEs/SAEs was higher with KR (27%) compared with R (15%), P=0.012; the most frequent were infections (KR 4% vs R 7%); all other events were reported in ≤5% of pts and included: gastrointestinal (KR 5% vs R 2%), cardiac (KR 4% vs R 1%), hypertension (KR 3% vs R 0%), and thrombotic microangiopathy (3% vs 0%). 4 pts developed a second primary malignancy in KR (breast 1 pt; thyroid 1 pt; myelodysplastic syndrome 1 pt; non-melanoma skin cancer 1pt) vs 1 pt in R (acute lymphoblastic leukemia). Dose reductions of R were reported in 23% of KR and 29% of R pts; dose reductions of K were reported in 20% of pts. The rate of discontinuation due to AEs was similar in the 2 arms (KR 10% vs R 9%). Conclusions. Treatment with KRd_ASCT significantly improved PFS compared with both KRd12 and KCd_ASCT. Maintenance with KR also improved PFS vs R. Figure Disclosures Gay: Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bristol-Myers Squibb: Honoraria, Membership on an entity's Board of Directors or advisory committees; Roche: Membership on an entity's Board of Directors or advisory committees; GSK: Membership on an entity's Board of Directors or advisory committees; AbbVie: Membership on an entity's Board of Directors or advisory committees; Adaptive: Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Membership on an entity's Board of Directors or advisory committees; Oncopeptides: Membership on an entity's Board of Directors or advisory committees; Janssen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees. Musto:Celgene: Honoraria; Amgen: Honoraria. Galli:BMS: Honoraria; Celgene: Honoraria; Janssen: Honoraria; Takeda: Honoraria. Belotti:Jannsen: Membership on an entity's Board of Directors or advisory committees; Amgen: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees. Zamagni:Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel, Accommodations, Expenses, Speakers Bureau; Celgene Corporation: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda: Honoraria, Other: Travel, Accommodations, Expenses, Speakers Bureau; Janssen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel, Accommodations, Expenses, Speakers Bureau; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel, Accommodations, Expenses, Speakers Bureau. Zambello:Janssen: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees. De Sabbata:Celgene: Membership on an entity's Board of Directors or advisory committees; Amgen: Membership on an entity's Board of Directors or advisory committees. D'Agostino:GSK: Membership on an entity's Board of Directors or advisory committees. Liberati:VERASTEM: Honoraria, Research Funding; ROCHE: Honoraria, Research Funding; PFIZER: Honoraria, Research Funding; ONCOPEPTIDES AB: Honoraria, Research Funding; TAKEDA: Honoraria, Research Funding; MORPHOSYS: Honoraria, Research Funding; ONCONOVA: Honoraria, Research Funding; ABBVIE: Honoraria, Research Funding; NOVARTIS: Honoraria, Research Funding; KARYOPHARM: Honoraria, Research Funding; INCYTE: Honoraria; JANSSEN: Honoraria; CELGENE: Honoraria; AMGEN: Honoraria; BMS: Honoraria; BEIGENE: Honoraria; ARCHIGEN: Honoraria; BIOPHARMA: Honoraria; FIBROGEN: Honoraria. Offidani:Janssen: Consultancy, Honoraria; BMS: Consultancy, Honoraria; Celgene: Consultancy, Honoraria. Cavo:AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; GlaxoSmithKline: Honoraria, Speakers Bureau; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel accomodations, Speakers Bureau; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Karyopharm: Honoraria; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel accomodations, Speakers Bureau. Boccadoro:AbbVie: Honoraria; Bristol-Myers Squibb: Honoraria, Research Funding; Novartis: Honoraria, Research Funding; Janssen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Honoraria, Research Funding; GlaxoSmithKline: Membership on an entity's Board of Directors or advisory committees; Mundipharma: Research Funding; Amgen: Honoraria, Research Funding; Sanofi: Honoraria, Research Funding. OffLabel Disclosure: The presentation includes discussion of off-label use of a drug or drugs for the treatment of multiple myeloma (including carfilzomib, cyclophosphamide, lenalidomide and dexamethasone).

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2020-136907

Language: English

Publisher: American Society of Hematology

Publication Date: 2020

detail.hit.zdb_id: 1468538-3

detail.hit.zdb_id: 80069-7

In: American Journal of Hematology, Wiley, Vol. 97, No. 2 ( 2022-02)

Type of Medium: Online Resource

ISSN: 0361-8609 , 1096-8652

URL: Issue

URL: Article

DOI: 10.1002/ajh.v97.2

DOI: 10.1002/ajh.26429

Language: English

Publisher: Wiley

Publication Date: 2022

detail.hit.zdb_id: 1492749-4

In: Blood, American Society of Hematology, Vol. 136, No. Supplement 1 ( 2020-11-5), p. 17-18

Abstract: Background. Patients with pre-existent chronic morbidities are likely to be more severely affected by SARS-Cov2 infection. In Italy, the "Società Italiana Talassemie ed Emoglobinopatie" (SITE) has recently estimated the number of patients (Pts) with Hemoglobinopathies followed by Italian Specialized Centers (SITE Network). Five thousand Transfusion-dependent beta-thalassemia (TDT), 1900 Non-Transfusion-dependent beta-thalassemia (NTDT) and 2000 Sickle Cell Disease (SCD) were registered [1]. To verify the impact of SARS-CoV-2 infection on Pts with Hemoglobinopathies, we performed a specific survey by electronic Case Report Form (eCRF). Inclusion criteria included positive swab or serology in a patient with hemoglobinopathy and at least 15 days of follow-up from either the onset of symptoms or SARS-CoV2 positivity. The survey was approved by the Ethics Committee, and eCRF was shared with the Centers of Italian Hemoglobinopathies Network. Preliminary data updated to April 10, 2020, were published [2] . Results. As of July 31, 2020, 27 cases have been reported: 18 TDT, 4 NTDT, 5 SCD. 89% of the cases were in Northern Italy, where the rate of infection was much higher than the rest of the country, reflecting the national epidemiology. The mean age of thalassemia patients (TDT and NTDT) was 43±11 years, and 55% were male; the mean age of SCD patients was 33±15 years, and 40% was male. The likely source of infection has been detected in 63% (17/27) of cases: 11 had occupational exposure, 6 had a positive relative. Five patients were asymptomatic: for them, the SARS-CoV-2 infection was identified by positive swab for 1 patient and by positive level of IgG for 4. Twenty patients had associated comorbidities, 14 were splenectomized, and 3 had functional asplenia. Eleven patients were hospitalized, only one in high-intensity care unit. Three patients required more intensive ventilation support with continuous positive airway pressure (CPAP), one of these has a history of diffuse large B-cell lymphoma treated with chemotherapy in the previous year. Three other patients required support by oxygen. No Pts required intubation. Two Pts increased blood requirement. Only five received supposedly specific treatment for COVID-19: two hydroxychloroquine (HCQ), one HCQ plus ritonavir/darunavir, and one HCQ plus anakinra, one HCQ plus Tocilizumab plus Lopinavir/Ritonavir. The clinical course of hospitalized patients was 18±7 days. All patients recovered. Conclusions. The prevalence of COVID-19 infection in Italian patients with Hemoglobinopathies result 0,3% while in general population the prevalence in Italy is 0,4% [3]. Considering that the thalassemia population is more strictly observed, we could postulate that the precautions suggested or self-applied by the Pts were effective. No death nor severe SARS with intubation, nor signs of cytokines storm, only one thromboembolic event was observed although most individuals had pre-existing complications. A single case with pulmonary hypertension has been described in detail [4] . In most individuals the infection has been pauci or asymptomatic and all recovered. This experience differs from what has been observed in Iran on a similar series with different severity and mortality and ask for a more in-depth comparison [5]. In conclusion, our data do not indicate increased severity of COVID-19 in Pts with Hemoglobinopathies followed in Specialized Centers. Acknowledgment. We would like to thank ALT (Associazione per la Lotta alla Talassemia R.Vullo - Ferrara).. References 1. http://www.site-italia.org/2020/covid-19.php. SITE communication. Accessed April 1, 2020 2. Motta I, Migone De Amicis M, Pinto VM, et al. SARS-CoV-2 infection in beta thalassemia: Preliminary data from the Italian experience. Am J Hematol. 2020;95(8): E198-E199. 3. https://www.epicentro.iss.it/coronavirus/sars-cov-2-dashboard, Accessed July 31, 2020 4. Pinto VM, Derchi GE, Bacigalupo L, Pontali E, Forni GL. COVID-19 in a Patient with β-Thalassemia Major and Severe Pulmonary Arterial Hypertension. Hemoglobin. 2020;44(3):218-220. 5. Karimi M, Haghpanah S, Azarkeivan A, et al. Prevalence and mortality in β-thalassaemias due to outbreak of novel coronavirus disease (COVID-19): the nationwide Iranian experience. Br J Haematol. 2020;190(3):e137-e140. Disclosures Motta: Sanofi Genzyme: Honoraria. Cappellini:BMS: Honoraria; Genzyme/Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees; CRISPR Therapeutics, Novartis, Vifor Pharma: Membership on an entity's Board of Directors or advisory committees. Piga:BMS: Research Funding; Novartis: Research Funding. Forni:Novartis: Membership on an entity's Board of Directors or advisory committees.

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2020-141610

Language: English

Publisher: American Society of Hematology

Publication Date: 2020

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detail.hit.zdb_id: 80069-7