Long-term efficacy and safety of 2CdA (cladribine) in extra-pulmonary adult-onset Langerhans cell histiocytosis: analysis of 23 cases from the French Histiocytosis Group and systematic literature review.

Langerhans cell histiocytosis (LCH) is a rare protean disease that usually affects children. Few data are available for management of adult-onset cases. A complete picture of the efficacy and safety of 2CdA (2-chlorodeoxyadenosine, cladribine) is lacking. We report a retrospective multicentre study of 23 adult LCH (a-LCH) patients who received single-agent 2CdA and a systematic literature review. All had previously received systemic therapy (vinblastine, n = 19). Response to 2CdA was evaluable in 22 cases. Overall response rate (ORR) was 91%. Complete response (CR) occurred in 11 cases (50%). Nine patients (39%) developed grade 3-4 neutropenia and/or severe infection. A literature review yielded 48 additional cases. A pooled analysis confirmed our findings (ORR: 88%, CR: 49%). CRs were rare with cumulative dose <50 mg/m2 . Disease progression rates were 20% and 30% at two and five years, respectively. Partial response (PR) to 2CdA was predictive of disease progression. Among eight re-treated patients, five went into CR, two in PR, and one died. Single-agent 2CdA is effective in reactivated a-LCH, including at intermediate doses. Toxicity, significant but acceptable, warrants infectious prophylaxis. Complete responders may enter prolonged remission. Further studies are needed to determine 2CdA sequencing with other agents (vinblastine, cytarabine).

Contribution and performance of mobile units in an organized mammography screening program.

BACKGROUND: The aims of this study were to evaluate the contribution of mobile mammography units to participation rate and to compare their performance to fixed screening centres within the organized mammography screening program of Quebec, Canada. METHODS: The study is based on all screening mammograms carried out in women aged 50-69 who participated in the Québec program from 2002 to 2010. Performance was measured by screening sensitivity, false-positive rate (1-specificity), positive likelihood ratio as well as abnormal call rate, detection rate, interval cancer rate, positive predictive value, and tumour characteristics. Poisson regression models with robust variance estimation were used to take into account the multi-level structure of the data. All models were adjusted for characteristics related to women. RESULTS: During the 2002-2010 period, 2,292,592 screening mammograms were performed, of which 42,279 (1.8%) were in mobile units. In regions serviced exclusively by mobile units, the participation rate reached an average of 63.4% during the 2006-2010 period compared to 54.7% for the entire study population. Estimated sensitivity was similar to that of fixed sites (rate ratio = 0.98 [0.84-1.15]) while the false-positive rate was lower (rate ratio = 0.76 [0.57-1.02]) although this difference was of marginal statistical significance (p=0.07). CONCLUSIONS: In this program, mobile mammography units allowed regions lacking a fixed centre to attain participation rates slightly higher than those in the rest of Quebec, without loss of sensitivity and with some gain in the false-positive rate.

Challenging the diagnosis of primary angiitis of the central nervous system: a single-center retrospective study.

OBJECTIVE: (1) To describe a series of adults assessed for suspected primary angiitis of the central nervous system (PACNS) and their final diagnosis; (2) to describe and compare presenting features of PACNS and reversible cerebral vasoconstriction syndrome (RCVS); and (3) to evaluate the specificity of the presenting features of RCVS. METHODS: Patients evaluated at our institution between 2000 and 2008 for a possible CNS vasculitis and investigated by conventional angiography and/or brain biopsy were retrospectively analyzed. The inclusion criteria were a clinicoradiological presentation and cerebral angiography and/or brain biopsy raising the hypothesis of isolated cerebral vasculitis; and absence of identifiable etiology at the time of conventional angiogram and/or brain biopsy. RESULTS: Among 58 cases evaluated, 37 met the inclusion criteria and 33 were included in the study. Thirteen patients had RCVS. Thunderclap headaches, the absence of a focal neurological deficit, a convexal subarachnoid hemorrhage and/or normal brain parenchyma on magnetic resonance imaging, and "string of beads" appearance on conventional angiography had high diagnostic value. Six patients had other noninflammatory vascular disorders (intracranial atherosclerosis, cryptogenic embolism, and genetic vasculopathy). Six patients had infection or malignancy. Eight patients were diagnosed with PACNS; their clinical presentation and disease course were heterogeneous. Brain biopsy was performed in 3 cases (positive in 1). CONCLUSION: RCVS is an important differential diagnosis of CNS vasculitis. Its particular presentation should allow rapid identification in order to avoid pointless investigations and treatment. The frequent lack of histological proof and heterogeneous presentation of PACNS illustrated the nosological uncertainties of this label.

Weekly home self-assessment of RAPID-4/3 scores in rheumatoid arthritis: a 6-month study in 26 patients.

OBJECTIVES: To investigate whether weekly determination of Routine Assessment of Patient Index Data (RAPID) scores 3 and 4 in patients with rheumatoid arthritis (RA) improved the assessment of disease activity and detected additional activity peaks (predictive of additional structural damage). METHODS: Each week for 6 months, 26 patients with RA completed the patient-reported outcome questionnaires RAPID-3 and RAPID-4. During the study period, the treatment regimen for RA remained unchanged in 23 of the 26 patients. RESULTS: RAPID-3 was as informative as RAPID-4. Mean values were 3.85±1.66 (range: 0.72-6.85) and 3.43±1.57 (range: 0.81-6.77), respectively. The areas under the RAPID-3 score curves plotted using only the first and last weeks or all the weeks showed a statistically significant difference in 19 (73%) of the 26 patients. The difference between the highest and lowest RAPID-3 scores was greater than the clinically significant threshold of 1.2 in all 26 patients (mean difference: 2.95±0.71; range: 1.6-5.5). In 13 patients, the RAPID-3 score detected one (one patient) or several (12 patients) activity peaks. Among RAPID-3 score components, the visual analog scale (VAS) pain score had the greatest influence (37% of the total score), followed by the VAS disease-activity score (36%) then by the multidimensional Health Assessment Questionnaire score (27%). Scores were not influenced by patient mood at questionnaire completion. CONCLUSION: Self-evaluation at home using the RAPID-3 score provides additional information that should improve the accuracy of RA monitoring between physician visits and that may help to optimize visit scheduling.