Patient-Reported Outcomes and Quality of Life Assessment. New Targets for New Targeted Therapy?

No abstract availabl

Long-Term observation of 208 adults with chronic idiopathic thrombocytopenic purpura

PURPOSE: TO define response to therapy and ultimate outcome of adults with idiopathic thrombocytopenic purpura (ITP). PATIENTS AND METHODS: We retrospectively analyzed patients with ITP diagnosed between 1978 and 1988, and reexamined them between June 1992 and March 1993. Data from 208 cases were collected. Median patient age was 44 years (range 14 to 78) at the time of diagnosis, and 51 years (range 19 to 86) at reexamination. Length of follow-up ranged from 48 to 151 months (median 92) and was longer than 10 years in 26 patients (12.5%). Reexamination included a careful interview, physical examination, complete blood count, screening for HIV infection, determination of platelet-bound IgG, and, in persistently thrombocytopenic patients, autoimmunity markers and routine laboratory investigations. RESULTS: A total of 121 patients with fewer than 50 x 10(9) platelets per liter received an initial treatment with prednisone (PDN) at a dosage of 1 mg/kg of body weight for 1 month. Refractory or relapsed cases underwent splenectomy and/or other therapeutic modalities. In 87 patients with greater than 50 x 10(9) platelets per liter, no therapy was scheduled. An initial complete response to PDN was observed in 38.8% cases. A sustained complete remission (CR) lasting more than 6 months with no maintenance therapy was attained in 18.7%. At the time of last follow-up only 11 of these patients remained in CR. Sixty-three patients underwent splenectomy. Forty-seven (74.6%) had a CR, with 41 achieving a prolonged recovery (>6 months). Twelve other cases attained a sustained partial remission. Long-lasting recoveries were observed in 7 other cases following alternative treatments. Spontaneous remissions occurred in 8 of 87 untreated cases after observation periods of 6 months or more. Eleven deaths were recorded (6 women and 5 men, median age 73), but only 5 were attributable to thrombocytopenia. At last control, 43 patients were in complete remission and free from therapy, and 52 were still on therapy. Four thrombocytopenic patients,had laboratory features and a clinical history consistent with an autoimmune disease. CONCLUSIONS: This analysis of ITP in adults suggests that splenectomy remains the most effective treatment. The majority of patients who undergo splenectomy can have a CR for many years, while only a minority of those who do not have this therapeutic modality or fail it are likely to attain similar results. The long-term prognosis of ITP is benign even in refractory cases. Spontaneous remissions can be observed in a significant percentage of untreated patients (about 9%). The development of overt autoimmune diseases is relatively uncommon. Particular attention should be given to the management of ITP in the elderly, where bleeding episodes of the central nervous system tend to occur more frequently

Extracorporeal photopheresis as first-line treatment in high-risk graft-versus-host disease

Background: Chronic graft versus host disease (cGVHD) is the major late complication after allogeneic stem cell transplantation. Standard therapy is steroid and Cyclosporine- A (CyA); however, immune suppression (ISS) related infections or unresponsiveness to ISS, are major mortality causes. Extracorporeal photopheresis (ECP) has shown activity in treatment of cGVHD, but its use has been limited to first-line-unresponsive cGVHD. Aim of the study. This is a single center pilot study testing feasibility of a programme of photopheresis in association with standard therapy as first line treatment in high risk cGVHD. High risk was defined as the presence of parameters predicting high cGVHD-related mortality. Secondary objectives were response and complications incidence. Patients: Among 9 pts fitting enrolling criteria, 2 refused due to logistic problem or low compliance with the procedure, 7 were enrolled. Median age was 40. Donor was HLA identical sibling in 6 cases and MUD in 1. All cases presented with extensive/moderate-severe cGVHD; Akpek score was > 0 in 3/7 pts. Treatment plan. Pts started with Prednison (PDN) 1 mg/kg and CyA at cGVHD diagnosis; ECP was started with a frequency of 4 application/month in the first 3 months and 2/month for the subsequent 9 months; PDN and CyA were slowly reduced until suspension, or otherwise modulated. Study duration was 1 year. Pts were ruled out the study in case of ECP suspension; requirement of other ISS drugs in case of GVHD progression unresponsive to standard therapy, or severe infections. Response was evaluated with standard criteria, as progression, partial response (PR), very good PR (vgPR) or complete response (CR). Results: Adherence to protocol was: 5/7 pts at 3 months, 4/7 at 6 and 9 months, 3/7 at 12 mm; exit from the study was due to infectious complications (2), ECP suspension due to venous access related thrombosis (1) and clear cGVHD progression (1). In evaluable pts, response (CR+very good PR / ≤PR) per trimester was 4/5, 2/4 and 3/4 at I, II and III respectively; at the IV trimester, 1 very good PR, 2 PR and 1 progression were observed. Complications were evaluated in 4 pts and are reported on the table. At 1 year, 2/7 pts died (1 TRM, 1 relapse). Conclusion: ECP in association with standard therapy is feasible; complications incidence seems to be similar to those observed in patients not treated with ECP; a larger group of patients is needed to evaluate response in this setting

Extracorporeal photopheresis as first-line treatment in high-risk graft-versus-host disease

Background: Chronic graft versus host disease (cGVHD) is the major late complication after allogeneic stem cell transplantation. Standard therapy is steroid and Cyclosporine- A (CyA); however, immune suppression (ISS) related infections or unresponsiveness to ISS, are major mortality causes. Extracorporeal photopheresis (ECP) has shown activity in treatment of cGVHD, but its use has been limited to first-line-unresponsive cGVHD. Aim of the study. This is a single center pilot study testing feasibility of a programme of photopheresis in association with standard therapy as first line treatment in high risk cGVHD. High risk was defined as the presence of parameters predicting high cGVHD-related mortality. Secondary objectives were response and complications incidence. Patients: Among 9 pts fitting enrolling criteria, 2 refused due to logistic problem or low compliance with the procedure, 7 were enrolled. Median age was 40. Donor was HLA identical sibling in 6 cases and MUD in 1. All cases presented with extensive/moderate-severe cGVHD; Akpek score was > 0 in 3/7 pts. Treatment plan. Pts started with Prednison (PDN) 1 mg/kg and CyA at cGVHD diagnosis; ECP was started with a frequency of 4 application/month in the first 3 months and 2/month for the subsequent 9 months; PDN and CyA were slowly reduced until suspension, or otherwise modulated. Study duration was 1 year. Pts were ruled out the study in case of ECP suspension; requirement of other ISS drugs in case of GVHD progression unresponsive to standard therapy, or severe infections. Response was evaluated with standard criteria, as progression, partial response (PR), very good PR (vgPR) or complete response (CR). Results: Adherence to protocol was: 5/7 pts at 3 months, 4/7 at 6 and 9 months, 3/7 at 12 mm; exit from the study was due to infectious complications (2), ECP suspension due to venous access related thrombosis (1) and clear cGVHD progression (1). In evaluable pts, response (CR+very good PR / ≤PR) per trimester was 4/5, 2/4 and 3/4 at I, II and III respectively; at the IV trimester, 1 very good PR, 2 PR and 1 progression were observed. Complications were evaluated in 4 pts and are reported on the table. At 1 year, 2/7 pts died (1 TRM, 1 relapse). Conclusion: ECP in association with standard therapy is feasible; complications incidence seems to be similar to those observed in patients not treated with ECP; a larger group of patients is needed to evaluate response in this setting

Rehabilitation need and referrals in hematopoietic stem cell transplantation. the experience of Quality of Life Working Party of the Rome Transplant Network

No abstract availabl