Treatment with short-term,high-dose cyclosporin A in children with refractory chronic idiopathic thrombocytopenic purpura

We report on 14 children (seven boys, seven girls) with chronic idiopathic thrombocytopenic purpura (ITP) refractory to multiple treatments, who were given a short-term therapy (range between 6 and 10 weeks) with high doses of cyclosporin A (CyA) (median, 10 mg/kg/d). Six patients experienced adverse events and one developed severe systemic mycosis during therapy. A complete response (CR) was observed in four patients and a partial response (PR) in three patients. Only the four CR patients, who were all girls, had a sustained response. These data suggest that CyA may be effective in some children with chronic symptomatic ITP.     

Accuracy of homogeneous models for photon diffusion in estimating neonatal cerebral hemodynamics by TD-NIRS

We assessed the accuracy of homogenous (semi-infinite, spherical) photon diffusion models in estimating absolute hemodynamic parameters of the neonatal brain in realistic scenarios (ischemia, hyperoxygenation, and hypoventilation) from 1.5 cm interfiber distance TD NIRS measurements. Time-point-spread-functions in 29- and 44-weeks postmenstrual age head meshes were simulated by the Monte Carlo method, convoluted with a real instrument response function, and then fitted with photon diffusion models. The results show good accuracy in retrieving brain oxygen saturation, and severe underestimation of total cerebral hemoglobin, suggesting the need for more complex models of analysis or of larger interfiber distances to precisely monitor all hemodynamic parameters.     

Local tumor recurrence after curative resection for rectal cancer

Local tumor recurrence rates after curative rectal cancer surgery with the end-to-end anastomosis stapler (EEA^®) are reportedly high. Therefore, a retrospective review in ten Yale-affiliated hospitals was undertaken to establish the outcome of surgical resection for rectal cancer in this patient population. Of those 373 patients who had had curative resections, 192 (52 percent) were abdominoperineal resections (APR); 105 patients (28 percent) had restorative resections with sutured anastomoses, and the EEA stapler was used in 76 patients (20 percent). There was an equal distribution of tumors in the various Dukes' stages in all three procedures. Local tumor recurrence was: APR 19 percent, SUT 17 percent, and EEA 24 percent, but local tumor recurrence was more frequent after EEA than APR for tumors 7 to 10 cm from the anal verge (32 vs. 13 percent, respectively,P<0.05), and the time to recurrence was least in EEA patients. It is concluded that local tumor recurrence is higher than expected for all three procedures and that the EEA stapler was associated with a greater risk of local tumor recurrence. These findings are attributed to surgeon-related technical operative factors rather than to the nature of the tumors themselves.     

Revision total knee arthroplasty: experience with tantalum cones in severe bone loss

Background

Tantalum-made cones have been developed to compensate for large bone defect involving metaphyseal segment or major portion of the condyle or plateau during knee revision prosthetic surgery.

Materials and methods

We present our experience with femoral and tibial tantalum cones in 11 knees (12 cones overall were used, 6 on femur and 6 on tibia) with 2B or 3 Engh defect type during knee revision arthroplasty. Both cemented and cementless cones were used. Patients were submitted to a specific study protocol in order to exclude a new or persistent infection. Only one intra-operative complication not related to the implantation of the trabecular metal cone was observed.

Results

Neither early nor late post-operative re-infection was reported in our series at a mean follow-up of 39.8 months. Radiological analysis showed no cases of aseptic loosening or migration of the components. The only post-operative complication was delayed union of the tibial tuberosity in a patient who required osteotomy for surgical exposure. All patients improved both clinically and functionally.

Conclusions

The results of this study support the use of femoral and tibial porous tantalum metaphyseal cones as a viable option for revision knee arthroplasty with large amount of bone defects in both tibia and femur. We think that the main advantages of tantalum cones compared to structural bone graft lie in faster full weight-bearing recovery and in the cones' potential long-term maintenance of mechanical support. By our results, we can eventually exclude any concern regarding a possible direct correlation of the trabecular metal cones with re-infection.     

Rituximab for the treatment of refractory autoimmune hemolytic anemia in children

Autoimmune hemolytic anemia (AIHA) in children is sometimes characterized by a severe course, requiring prolonged administration of immunosuppressive therapy. Rituximab is able to cause selective in vivo destruction of B lymphocytes, with abrogation of antibody production. In a prospective study, we have evaluated the use of rituximab for the treatment of AIHA resistant to conventional treatment. Fifteen children with AIHA were given rituximab, 375 mg/m(2)/dose for a median of 3 weekly doses. All patients had previously received 2 or more courses of immunosuppressive therapy; 2 patients had undergone splenectomy. After completing treatment, all children received intravenous immunoglobulin for 6 months. Treatment was well tolerated. With a median follow-up of 13 months, 13 patients (87%) responded, whereas 2 patients did not show any improvement. Median hemoglobin levels increased from 7.7 g/dL to a 2-month posttreatment level of 11.8 g/dL (P <.001). Median absolute reticulocyte counts decreased from 236 to 109 x 10(9)/L (P <.01). An increase in platelet count was observed in patients with concomitant thrombocytopenia (Evans syndrome). Three responder patients had relapse, 7, 8, and 10 months after rituximab infusion, respectively. All 3 children received a second course of rituximab, again achieving disease remission. Our data indicate that rituximab is both safe and effective in reducing or even abolishing hemolysis in children with AIHA and that a sustained response can be achieved in the majority of cases. Disease may recur, but a second treatment course may be successful in controlling the disease.     

Endocrine function and bone disease during long‐term chelation therapy with deferasirox in patients with β‐thalassemia major

Iron overload in β‐thalassemia major (TM) typically results in iron‐induced cardiomyopathy, liver disease, and endocrine complications. We examined the incidence and progression of endocrine disorders (hypothyroidism, diabetes, hypoparathyroidism, hypogonadism), growth and pubertal delay, and bone metabolism disease during long‐term deferasirox chelation therapy in a real clinical practice setting. We report a multicenter retrospective cohort study of 86 transfusion‐dependent patients with TM treated with once daily deferasirox for a median duration of 6.5 years, up to 10 years. No deaths or new cases of hypothyroidism or diabetes occurred. The incidence of new endocrine complications was 7% (P = 0.338, for change of prevalence from baseline to end of study) and included hypogonadism (n = 5) and hypoparathyroidism (n = 1). Among patients with hypothyroidism or diabetes at baseline, no significant change in thyroid parameters or insulin requirements were observed, respectively. Mean lumbar spine bone mineral density increased significantly (P < 0.001) and the number of patients with lumbar spine osteoporosis significantly decreased (P = 0.022) irrespective of bisphosphonate therapy, hormonal replacement therapy, and calcium or vitamin D supplementation. There were no significant differences in the number of pediatric patients below the 5th centile for height between baseline and study completion. Six pregnancies occurred successfully, and four of them were spontaneous without ovarian stimulation. This is the first study evaluating endocrine function during the newest oral chelation therapy with deferasirox. A low rate of new endocrine disorders and a stabilization of those pre‐exisisting was observed in a real clinical practice setting. Am. J. Hematol. 89:1102–1106, 2014. © 2014 Wiley Periodicals, Inc.