ALBERT

Description: Introduction: Acquired hemophilia is a rare bleeding disorder with significant mortality risk but is yet highly treatable. Patients diagnosed with this condition in our institution have been treated with a standardized approach aimed at detecting and controlling bleeding early and inhibitor elimination. Objective: Review the effectiveness and treatment outcome of our therapeutic approach to acquired hemophilia patients. Methods: Consecutive patients diagnosed with acquired hemophilia over a period of 5 years from July 1998 till July 2004 were retrospectively reviewed for their underlying characteristics, responses to treatment, complications and subsequent outcome. Results: 13 patients were recorded with age ranging from 17 to 89 years (median 66 years) and a male to female ratio of 10:3. Follow-up periods range from 5 to 56 months. All patients presented with variable degree of bleeding. Four patients were given recombinant factor VIIa (from 1 to 5 doses) for the control of life threatening bleeding. Three patients received prothrombin complexes as bypassing agents. One other patient received both agents..All patients received both cyclophosphamide (50–100mg/day) and prednisolone (1 mg/kg/day) for elimination of inhibitors at diagnosis. Six patients received intravenous immunoglobulin of 1 gm/kg/day for 2 days with this decision based on the severity of bleeding. Two early deaths were recorded from retroperitoneal bleeding and cerebral infarct following recombinant factor VIIa therapy, respectively. Another death occurred following sepsis with multiple co-morbidities, while being given immunosuppressive therapy. He was the only patient experiencing significant leucopenia while on cyclophosphamide therapy. Two patients were lost to follow-up subsequently. Of 8 patients evaluable for response to inhibitor eliminating therapy with cyclophosphamide and prednisolone, all but one achieved a complete remission within 7 to 71 days with a median of 41 days. Of these 7 patients, there was one early relapse, which occurred from non-compliance to treatment while it was being tailed down. Remission was successfully reinduced on reintroduction of both cyclophophamide and prednisolone. All other patients have remained in remission after discontinuation of therapy. The patient with failure of complete response had persistent low inhibitor levels without bleeding complications and died after 56 months of follow up from unrelated causes. Overall mortality of evaluable patients was 27% (3 out of 11). Conclusion: Mortality remains significant in this condition with control of early bleeding risks and treatment complications paramount to survival. For the great majority of patients, our strategy affords effective inhibitor elimination and sustained remission of this condition

Description: PURPOSE: To evaluate the activity of low dose dexamethasone (Dex) and thalidomide (Thal) plus higher frequency zoledronic acid (Zol) - dtZ - in patients with symptomatic multiple myeloma (MM) who are unable to tolerate standard doses of Dex and/or Thal and/or chemotherapy. PATIENTS AND METHODS: Eighteen consecutive previously treated patients with symptomatic MM who were unable to tolerate conventional doses of Dex and/or Thal and/or chemotherapy were treated (on an intention to treat basis) with a low-toxicity regimen, dtZ; which comprises weekly Dex 20 mg OM for 4 days, Thal 50 mg ON, and three-weekly Zol 4 mg. Patients were treated for at least 3 months, and for up to 27.5 months. Five patients had complex karyotypes; including two patients with chromosome 13 deletion. The reasons for intolerance to standard therapy included pancytopenia, active viral hepatitis B, severe cardiovascular disease and recurrent severe infections. RESULTS: The response rate was 78%, including good responses in 61%, and complete remissions in 22% of patients. All five patients with complex karyotypes showed good response to dtZ. The median time to remission was 8.2 months. Grade 2 toxicity included motor neuropathy (two patients) and infection (one patient); and grade 3 toxicity included motor neuropathy (two patients) and infection (one patient). No cases of deep venous thrombosis or pulmonary embolism were reported. All patients received calcium/vitamin D supplements; and mild, transient hypocalcemia was reported in only two patients. These were rapidly corrected with intravenous calcium gluconate. Mild, transient hepatitis was observed in two patients, which resolved spontaneously. Patients with active hepatitis B received lamivudine concurrently with no adverse effects. Contrary to expectation, more frequent dosing of Zol was associated with overall improved renal function (p