Myotonic dystrophy is an autosomal dominant muscle disorder characterized by muscle wasting and weakness and a number of other systemic abnormalities. Some patients have hypo-IgG that is asymptomatic ...in most of them. We report the case of a 42-year-old woman with myotonic dystrophy and hypo-IgG who experienced asthenia and weight loss secondary to Giardia lamblia bowel infection.
Objective
Data on sustained remission of granulomatosis with polyangiitis (GPA) after discontinuation of therapy (referred to as GPA with sustained remission off‐therapy SROT) are scarce. In the ...present study, SROT among GPA patients from the French Vasculitis Study Group Registry was evaluated to identify factors associated with its occurrence and durability.
Methods
For inclusion of patients in the study, the diagnosis of GPA had to meet the GPA classification criteria defined by the American College of Rheumatology and/or the revised Chapel Hill Consensus Conference nomenclature for vasculitis. SROT was defined as achievement of remission (a Birmingham Vasculitis Activity Score of 0) that was sustained for ≥6 consecutive months after having discontinued glucocorticoid (GC) and immunosuppressant treatments. The characteristics of the patients at baseline and treatments received were compared at 3, 5, and 10 years postdiagnosis according to whether or not SROT had been reached and maintained.
Results
Among 795 patients with GPA, 92 GPA patients with SROT at 3 years postdiagnosis were compared to 342 control subjects who had experienced disease relapse and/or were still receiving GCs or immunosuppressants. No baseline differences were found, but patients with SROT at 3 years postdiagnosis had more frequently received intravenous cyclophosphamide as induction therapy compared to control subjects (P = 0.01), with a higher median number of infusions (P = 0.05). At 5 years postdiagnosis, no baseline differences were observed between groups, but patients with SROT at 5 years postdiagnosis had received more cyclophosphamide infusions compared to control subjects (P = 0.03). More patients with SROT had received rituximab as maintenance therapy than control subjects at 3 years and 5 years postdiagnosis (P = 0.09 and P < 0.001, respectively). Of the 74 patients enrolled in the GPA Registry with 10‐year follow‐up data after having received conventional maintenance therapy, 15 (20%) had reached SROT at 3 years, and 5 (7%) maintained SROT at 10 years postdiagnosis.
Conclusion
After conventional therapies, 7% of GPA patients had reached SROT at 10 years postdiagnosis. No baseline vasculitis characteristics distinguished patients who achieved/maintained SROT from those who experienced disease relapse and/or those who continued to receive GCs or immunosuppressant therapy, but patients with SROT had received more intensive induction therapy and rituximab as maintenance therapy more frequently.
Background:
There is no standard treatment for R/RMCL patients who have failed first line treatment. With an overall response rate (ORR) of 78 %, and a median PFS of 15,3 months ibrutinib appears as ...one of the best treatment in this setting (Rules et al, BJH, 2017). At the ASH 2016 meeting we have presented the data of 49 R/R MCL patients treated by the RiVBD combination (Rituximab‐Bendamustine‐Bortezomib‐Dexametasone) previously described in first line (Gressin et al, Haematologica, 2018). Here we present our updated data, with a larger cohort and a longer follow up.
Aims:
To determine the efficacy and safety of the RiBVD regimen in the salvage therapy of R/R MCL patients.
Methods:
We proposed to all LYSA partner centers a survey to retrospectively evaluate the efficacy of the RiBVD regimen in R/R MCL patients in second line or more than 2 lines of treatment. The RiBVD regimen comprises: Rituximab 375 mg/sqm IV D1, Bendamustine 90 mg/sqm IV D1 and D2, bortezomib 1,3 mg/sqm SC D1, D4, D8, D11 and dexamethasone 40 mg IV D2. The response was evaluated by the Cheson criteria 2007 with or without PET scan. ORR, CR, and survival (PFS, DOR and OS) were calculated.
Results:
67 patients from 19 centers were recruited between June 2016 and January 2019. Nineteen patients (28%) were diagnosed with a blastoid variante. Thirty‐one patients were refractory (46%) to their previous line, 91% (61/67) had received at diagnosis a regimen including high dose cytarabine. The median age at D1 of RiBVD was 69 years (40–91y), 28% (n = 19) were young (<65y) and 72% (n = 48) older patients (>65y).
Treatment history: Thirty‐three patients (49%) received RiBVD in second line, 16 in third line and 18 after the third line. In second line, 28/33 (85%) patients had received previously high dose cytarabine,76 % (25/28) were refractory and 54% were old patients. Twelve patients received RiBVD after a failure of an ibrutinib treatment.
Toxicities: 27 patients (40%) have presented a grade III/IV hematological toxicities with 9 septic shock but no death related toxicities. Three patients died in CR (1 patient developped MDS and 2 for public road accident)
Efficacy: The ORR of the 67 patients was 71% (48/67), 32 were in CR (48%) and 16 in PR. For patients treated in 2nd line, the ORR was 82% (27/33) with 20 CR (60%) and 7 PR. After the second line ORR was 62% (21/34) with 11 CR (52%). The ORR of relapsed pts were 83% (30/36 with 22 CR and 8 PR) and refractory pts were 58% (18/31 with 11 CR and 7 PR). For the 19 blastoid variante the ORR and CR were respectively 58% and 36,8%. Regarding RiBVD after ibrutinib, the ORR was 66% (8/12 with 3 CR and 5 PR) and the DOR was 17 months. With a 21.2 months median follow‐up, the median OS of all patients was 32 months, the median PFS was 10,1 months and the DOR was 18 months. The main results are summarized in Table 1.
Summary/Conclusion:
The RiBVD regimen appears as one of the best option as salvage therapy of R/R MCL after a cytarabine based regimen either after ibrutinib resistance. The major prediction of responses are the first relapse and the classic form.
