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FDA approves first treatment for rare disease in patients who receive stem cell transplant from blood or bone marrow

The U.S. Food and Drug Administration today approved Defitelio (defibrotide sodium) to treat adults and children who develop hepatic veno-occlusive disease (VOD) with additional kidney or lung abnormalities after they receive a stem cell transplant from blood or bone marrow called hematopoietic stem cell transplantation (HSCT). This is the first FDA-approved therapy for treatment of severe hepatic VOD, a rare and life-threatening liver condition.
Source: Food and Drug Administration - Category: American Health Source Type: news

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Abstract Calciphylaxis is a rare disease characterized by calcification of small- to medium-sized blood vessels in the dermis and subcutaneous fat, resulting in cutaneous necrosis. Although most commonly shown in patients with end-stage kidney disease, it has also been reported in patients with other diseases, including alcoholic cirrhosis and malignancies. Here, we report an unusual case of calciphylaxis in an orthotopic liver transplant recipient with acute kidney injury. The patient, a 43-year-old white female with a history of type 2 diabetes mellitus, alcoholic cirrhosis, and normal kidney function, presented...
Source: Experimental and Clinical Transplantation : official journal of the Middle East Society for Organ Transplantation - Category: Transplant Surgery Authors: Tags: Exp Clin Transplant Source Type: research
Atypical hemolytic-uremic syndrome (aHUS) is an extremely rare disease, and up to 70% of the patients have a genetic mutation in the encoding components of complement activation or anti-complement factor H autoantibodies. The risk of recurrence after kidney transplantation is 10% to 80%. Eculizumab, a monoclonal antibody that binds complement protein C5, has shown to be highly effective in patients with aHUS; however, there are only few reports on the efficacy and safety of long-term eculizumab treatment in children with recurrent aHUS.
Source: Transplantation Proceedings - Category: Transplant Surgery Authors: Tags: Original Contributions: Case Reports Source Type: research
We report a case of partial cystectomy for urinary bladder paraganglioma before living kidney transplantation.
Source: Transplantation Proceedings - Category: Transplant Surgery Authors: Tags: Original Contributions: Case Reports Source Type: research
Post kidney transplantation progressive multifocal leukoencephalopathy (PML) is a rare disease on which there are very few published reports on record. PML is a demyelinating disease caused by a destructive infection of the oligodendrocytes by the JC polyomavirus. No effective therapeutic protocol has been established other than measures to revive the immune function by reducing or discontinuing the administration of immunosuppressive agents. Most cases are progressive and show a poor prognosis.
Source: Transplantation Proceedings - Category: Transplant Surgery Authors: Source Type: research
We report three cases; all referred after birth with lax abdominal wall, congenital anomalies of kidney, and urinary tract. One of the patients had an absent right foot. They all had cryptorchidism, and in one, there was deranged renal function. The reported cases had both medical and radiological interventions to varying degrees. They all had an abdominal ultrasound which revealed varying degrees of hydronephrosis, hydroureters, and bladder changes. Voiding cystourethrogram showed vesicoureteric reflux in one of the reported cases. Urinary tract infections were appropriately treated with antibiotics based on sensitivity. ...
Source: Saudi Journal of Kidney Diseases and Transplantation - Category: Urology & Nephrology Authors: Tags: Saudi J Kidney Dis Transpl Source Type: research
CONCLUSION: In patients with aHUS associated with a CFH mutation, KT without LT was complicated with a recurrence of aHUS, which might lead to allograft loss. Conversely, LT was successful in preventing the recurrence of aHUS and thus might be another option for a recurrence-free life for aHUS patients associated with CFH mutation. PMID: 29215813 [PubMed - in process]
Source: J Korean Med Sci - Category: General Medicine Authors: Tags: J Korean Med Sci Source Type: research
We report a patient with a diagnosis of AS who developed end-stage kidney disease (ESKD) and received a kidney transplant from a living unrelated donor. The graft biopsy specimen surprisingly showed a pattern of thin basement membranes.
Source: Transplantation Proceedings - Category: Transplant Surgery Authors: Tags: Original Contributions: Case Reports Source Type: research
Thrombotic microangiopathy (TMA) is a serious complication of transplantation that adversely affects kidney transplant recipient and allograft survival. Post-transplant TMA is usually classified into two categories: 1) recurrent TMA and 2) de novo TMA. Atypical hemolytic uremic syndrome (aHUS) resulting from dysregulation and over-activation of the alternate complement pathway is a rare disease but the most common diagnosis associated with recurrence in the allografts. De novo TMA, on the other hand, represents an overwhelming majority of the cases of post-transplant TMA and is a substantially more heterogeneous entity than recurrent aHUS.
Source: Transplantation Reviews - Category: Transplant Surgery Authors: Tags: Review article Source Type: research
A single center kidney transplant experience among ten Caucasian females with end-stage renal disease due to scleroderma
. Clin Nephrol. 2017 Apr 13;: Authors: Saxena N, Djamali A, Astor BC, Mohamed M, Mandelbrot D, Parajuli S Abstract There is limited information on kidney transplant recipients with end-stage renal disease (ESRD) due to scleroderma. We conducted an observational study on kidney transplant recipients with ESRD due to scleroderma who received kidney transplant at our center between 01/1994 and 06/2013. During the study period, there were 10 kidney transplant recipients, all of whom w...
Source: Clinical Nephrology - Category: Urology & Nephrology Authors: Tags: Clin Nephrol Source Type: research
David Fajgenbaum has nearly died five times. He’s had his last rites read to him, and he knows he could relapse at any moment. Still, the 32-year-old doctor doesn’t waste energy on self-pity. He can’t. He’s too busy trying to find a cure for the rare disease that’s come so close to killing him. “I realized that if I didn’t dedicate the rest of my life to trying to cure this disease, that no one else was going to do it,” Fajgenbaum said. “I didn’t have many more shots.” When Fajgenbaum was 25 and in his third year at the Perelman School of Medicine at t...
Source: Healthy Living - The Huffington Post - Category: Consumer Health News Source Type: news
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