Patient consultation about a trial of therapeutic plasma exchange for complex regional pain syndrome.

BACKGROUND: Complex regional pain syndrome (CRPS) is a severe post-traumatic chronic pain condition affecting distal limbs, for which few effective treatments exist. Complex regional pain syndrome is listed in the 2016 American Society for Apheresis guidelines as an indication for plasma exchange treatment, but patient perspectives are lacking. STUDY DESIGN AND METHODS: We convened a "patient and public consultation exercise." Supervised by a clinical ethicist, the case for using therapeutic plasma exchange (TPE) was presented by a researcher and two TPE experts to five patients with severe, long-standing CRPS and to one relative. Discussions were recorded and transcribed. RESULTS: Participants supported the technology's use but expressed concern that the small trauma of repeat cannulations of CRPS unaffected limbs might theoretically cause a spread of the condition, a risk which requires highlighting when taking consent. For a preliminary trial, the participants proposed to include no less than 10, preferably 20 participants. They suggested that the threshold for a decision to conduct a definite trial based on preliminary trial results should be set no higher than 1/5 patients achieving >30% pain reduction in the preliminary trial, with half of these responders achieving >50%. The use of sham-TPE and a long trial duration (1 year) of a definite, parallel trial was considered acceptable, provided patients would be offered voluntary swap to the other trial arm at the end of the main trial period. CONCLUSION: These results provide pertinent patient views about TPE treatment which can inform both clinical consultation and consent procedure and the design of future trials.

BCR-ABL peptide vaccination in healthy subjects: immunological responses are equivalent to those in chronic myeloid leukaemia patients.

We and others have reported that vaccination of chronic myeloid leukaemia (CML) patients with e14a2 BCR-ABL junctional peptides can elicit moderate but transient T cell responses. To determine whether CML patients may be tolerised to BCR-ABL, here we used the same schedule to vaccinate 5 healthy subjects. Although IFN-γ and granzyme-B production, and proliferative responses to the vaccine peptides were detected in all 5 cases, responses were statistically similar to CML patients. CML patients are therefore not appreciably tolerised to BCR-ABL, and junctional peptides may only be moderately immunogenic, underlining the importance of antigen immunogenicity when designing vaccination strategies.

Gaucher disease and multiple myeloma.

Gaucher disease (GD) is the most frequent lysosomal storage disease and corresponds to an inherited deficiency of glucocerebrosidase. Due to excessive accumulation of glucocerebroside in bone marrow, both cytopenia and bone lesions may occur. The incidence of malignant disorders has been evoked in non-neuronopathic type I GD. More particularly, many case reports have been published that describe the association between GD and multiple myeloma (MM). Here, we first deal with diagnosis criteria that allow to distinguish between bona fide Gaucher celles and the so-called pseudo or pseudo-pseudo Gaucher cells. We then analyse relevant case reports and recent articles that provide convincing data regarding GD and MM association and suggest physiopathological links between the two disorders.