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PubMed2006

Safety and efficacy of risk-adapted cyclophosphamide, thalidomide, and dexamethasone in systemic AL amyloidosis.

High-dose melphalan with stem-cell transplantation is believed to be the most effective treatment for systemic light-chain (AL) amyloidosis, but many patients are ineligible because of the extent of their disease, and treatment-related mortality (TRM) remains substantial. We report the use of a risk-adapted oral regimen of cyclophosphamide, thalidomide, and dexamethasone (CTD) or attenuated CTD (CTDa) in 75 patients with advanced AL amyloidosis, including 44 patients with clonal relapse after prior therapy. Fifty-one (68%) patients received CTD and 24 (32%) received CTDa. A hematologic response occurred in 48 (74%) of 65 evaluable patients, including complete responses in 14 (21%) and partial responses in 34 (53%) cases. Median estimated overall survival (OS) from commencement of treatment was 41 months, and from diagnosis median was not reached with a median follow-up of 22 months. Three-year estimated OS was 100% and 82% among complete and partial hematologic responders, respectively. Toxicity necessitating cessation of therapy occurred in 8% and was at least grade 2 in 52% of patients. TRM was 4%. The clonal response rates to CTD reported here are higher than any previously reported nontransplantation regimen in AL amyloidosis, and risk adaptation allows its use in poorer risk patients. CTD merits prospective randomized study.

Administration, Oral↗
PubMed2001

Acquired factor X deficiency in patients with amyloid light-chain amyloidosis: incidence, bleeding manifestations, and response to high-dose chemotherapy.

Acquired deficiency of factor X occurs in patients with systemic amyloid light-chain (AL) amyloidosis, presumably due to adsorption of factor X to amyloid fibrils. Of 368 consecutive patients with systemic AL amyloidosis evaluated at Boston Medical Center, 32 patients (8.7%) had factor X levels below 50% of normal. Eighteen of these patients (56%) had bleeding complications, which were more frequent and severe in the 12 patients below 25% of normal; 2 episodes were fatal. Ten factor X-deficient patients received high-dose melphalan chemotherapy followed by autologous stem cell transplantation. Of 7 patients alive 1 year after treatment, 4 had a complete hematologic response, and all 4 experienced improvement in their factor X levels. One of 2 additional patients with partial hematologic responses had improvement in factor X. Thus, aggressive treatment of the underlying plasma cell dyscrasia in AL amyloidosis can lead to the amelioration of amyloid-related factor X deficiency.

Amyloid↗
PubMed1999

Prospective randomized trial of melphalan and prednisone versus vincristine, carmustine, melphalan, cyclophosphamide, and prednisone in the treatment of primary systemic amyloidosis.

PURPOSE: Primary systemic amyloidosis is an immunoglobulin deposition disorder in which insoluble light chains cause organ dysfunction and death. The established conventional therapy is treatment with melphalan and prednisone. We investigated whether treatment with multiple alkylating agents improved the response rate or survival time, compared with melphalan and prednisone therapy. PATIENTS AND METHODS: We treated 101 patients with biopsy-proven primary amyloidosis. The patients were randomly assigned to receive melphalan and prednisone (52 patients) or vincristine, carmustine, melphalan, cyclophosphamide, and prednisone (49 patients). Patients were stratified according to the presence of cardiac involvement, time from diagnosis to randomization, serum beta2-microglobulin level, and whether peripheral neuropathy was the major manifestation of the disease. RESULTS: The median duration of survival after randomization was 29 months, with no differences in survival time between the two groups. There were 29 patients who fulfilled the response criteria: 15 in the vincristine, carmustine, melphalan, cyclophosphamide, and prednisone arm and 14 in the melphalan and prednisone arm. CONCLUSION: Therapy with multiple alkylating agents did not result in a higher response rate or longer survival time, compared with standard melphalan and prednisone treatment in patients with primary systemic amyloidosis.

Adult↗
PubMed1988

Thyroid amyloidosis with recurrent subacute thyroiditis-like syndrome.

We studied 2 men with a subacute thyroiditis-like syndrome (STLS) associated with systemic amyloidosis. Both had very tender, diffuse, firm goiters, low thyroidal radioactive iodine uptake values, and increased erythrocyte sedimentation rates. Glucocorticoid therapy resulted in dramatic improvement. Compared to 18 patients with subacute thyroiditis, these 2 men had 1) persistence of goiter even in remission, 2) repeated exacerbation of STLS, 3) pain always localized in the same site, and 4) gastrointestinal, renal, and cardiac abnormalities. Histological examination of the patients' thyroid glands revealed amyloid deposition and no evidence of subacute thyroiditis. In addition, 1 man had low T3 thyrotoxicosis with an elevated rT3/T3 ratio, suggesting impaired peripheral conversion of T4 to T3, and immunological and histological evidence of Hashimoto's thyroiditis. These findings suggest that thyroid amyloidosis may be associated with STLS. When patients with clinical features of subacute thyroiditis have an unusual course, the possibility of thyroid amyloidosis should be considered.

Adult↗
PubMed1999

Transthyretin amyloidosis and superficial siderosis of the CNS.

OBJECTIVE: To describe a previously unreported clinical and radiologic presentation of hereditary transthyretin (TTR)-related amyloidosis. BACKGROUND: Unexplained cerebellar ataxia, pyramidal syndrome, and hearing loss are observed in some patients with TTR-related amyloidoses. METHODS: We performed clinical, radiologic, and pathologic examinations of three family members with TTR-related (Ala36Pro) amyloidosis. RESULTS: The patient was a 69-year-old woman with vitreal amyloid deposits, progressive sensorineural deafness, cerebellar ataxia, pyramidal syndrome, and recurrent transient neurologic symptoms. Cranial MRI showed symmetric thin rims of low signal intensity in T2- and T2*-weighted images in the cortex of the sylvian fissures, of the cerebellar hemispheres and vermis, and in the quadrigeminal plate consistent with superficial siderosis of the CNS. Her older daughter had vitreal amyloid deposits, acute Brown-Sequard syndrome at C4, acute sensorineural deafness, and recurrent transient neurologic symptoms. Cranial MRI at age 48 revealed a rim of low signal intensity in T2- and T2*-weighted images in the superior vermis folia and the right sylvian cortex. In addition, two small hemosiderin deposits were seen in the left parietal cortex. Lumbar puncture yielded colorless CSF with increased ferritin content and was followed by fourth ventricle hemorrhage. Cranial MRI 11 months later showed progression of brain hemosiderin deposits. The younger daughter had vitreal deposits, sensorimotor polyneuropathy, and acute sensorineural hearing but no evidence of siderosis on cranial MRI. She died at age 43 years of posterior fossa subarachnoid hemorrhage, and the neuropathologic examination showed amyloid deposition in the leptomeningeal spaces and vessels. CONCLUSION: Transthyretin-related amyloidosis may cause superficial siderosis of the CNS through subarachnoid bleeding related to meningovascular amyloid deposition.

Adult↗
PubMed1991

Musculoskeletal manifestations of amyloidosis. A review of 83 patients on haemodialysis for at least 10 years.

Long-term regular haemodialysis for chronic renal failure is associated with amyloidosis. In this condition excess amounts of the unexcretable plasma protein beta-microglobulin are laid down in tendons, joints and bones. Amyloidosis presents with various musculoskeletal disorders only after several years of dialysis. We reviewed 83 patients who had been dialysed for at least 10 years. The commonest complaint was severe joint pain in the absence of radiological changes of arthritis (41%), the shoulders usually being the most affected (33%). Carpal tunnel syndrome had developed in 26 patients, and was bilateral in 14 of them; at operation the presence of amyloid was confirmed. Six of these patients had recurrent symptoms after a further two to three years and required another decompression. Other manifestations of amyloidosis included trigger finger, flexor tendon contracture, spontaneous tendon rupture and pathological fracture through amyloid bone cysts. The frequency of symptoms was proportional to the duration of dialysis: all 13 patients on dialysis for over 20 years were affected. Symptoms developed earlier in older patients.

Adolescent↗
PubMed1977

Coincidence of sarcoidosis and amyloidosis.

Amyloidosis was diagnosed in a patient with sarcoidosis. There was no evidence of any other disease known to be associated with amyloidosis. The joint occurrence of amyloidosis and sarcoidosis appears to be extremely rare, and to our knowledge, this is the second such patient to be reported. Whether the association of the two diseases in this patient represents a causal relationship remains an open question.

Amyloidosis↗
PubMed1979

Pulmonary amyloidoma and hilar adenopathy. Rare manifestations of primary amyloidosis.

Three cases of primary intrathoracic amyloidosis are reported. Two of these three patients initially had localized parenchymal pulmonary amyloid tumors (or pulmonary amyloidomas). The third patient developed bilateral enlargement of the hilar lymph nodes as a manifestation of primary amyloidosis. These rare manifestations of primary amyloidosis are illustrated, and their clinical signifiance is discussed. Amyloidoma should be considered in patients with unexplained pulmonary nodules or enlargement of hilar lymph nodes.

Aged↗
PubMed1990

Amyloidosis and pleural disease.

Pleural involvement with systemic amyloidosis has been reported rarely in the literature. Diagnosis of this entity by percutaneous needle biopsy of the pleura has been described only in two prior case reports. We describe five patients in whom the diagnosis of pleural amyloidosis was established by Cope needle biopsy during evaluation of pleural effusions of indeterminate cause. Three patients presented with a history suggestive of multiorgan disease and a pleural biopsy performed despite a transudative effusion demonstrated amyloid infiltration of the pleura, obviating the need for other organ biopsies. We conclude that in patients with pleural effusions, if history suggests multiorgan involvement and there is suspicion for amyloidosis, then a closed pleural biopsy with special stains for amyloid should be performed even if the effusions are transudative. This may be the diagnostic procedure of choice in such patients.

Aged↗
PubMed2000

Novel approaches to the treatment of primary amyloidosis.

Primary (AL, amyloid light-chain) amyloidosis is a plasma cell disorder in which deposits of amyloid light-chain protein cause progressive organ failure. It is important to recognise that amyloidosis is a dynamic process and chemotherapy-induced reduction of the activity of the plasma cell clone reduces the supply of the amyloid precursor protein and can result in a major regression of the deposits. The most common target organ is the kidney and renal amyloidosis manifests as proteinuria or nephrotic syndrome. Proteinuria is seen in three quarters of patients. Amyloid related nephrotic syndrome and renal failure are potentially reversible. Fatigue, congestive heart failure, hepatomegaly, peripheral neuropathy, orthostatic hypotension, carpal tunnel syndrome and macroglossia are other common features. The median survival is one to two years. Conventional-dose melphalan as standard treatment can prolong the median duration of survival by about ten months, but the clinical response rates with improvement of impaired organ function are low. Up-front high-dose chemotherapy with autologous peripheral blood stem cell transplantation is much more effective and can result in a major improvement in the clinical condition of patients. However, the toxicity related to this treatment can be relevant due to impaired organ function. Conventional-dose chemotherapy consisting of vincristine, doxorubicin and dexamethasone or high-dose dexamethasone or interferon-alpha are other possible approaches to treatment. The improvement of patient condition with an effective conventional-dose chemotherapy may increase the tolerability of high-dose chemotherapy and reduce transplantation related problems.

Amyloidosis↗
PubMed2004

Axoval neuropathy as initial manifestation of primary amyloidosis: report of a case submitted to bone marrow transplantation.

Amyloidosis is a syndrome characterized by deposition of a highly insoluble protein material in the extracellular space that may affect several organs. It may be generalized and idiopathic (primary amyloidosis). We describe the case of a 48 years-old woman with axonal neuropathy associated with proteinuria, whose final investigation resulted in diagnosis of primary amyloidosis (AL). She was submitted to autologous bone marrow transplantation. We discuss some aspects related to diagnosis of neuropathy and current treatment of AL.

Amyloid Neuropathies↗
PubMed2006

Serum cardiac troponin T in cardiac amyloidosis: serial observations in five patients.

The most common cause of death of patients suffering from amyloidosis is heart failure and sudden death, and cardiac troponin T (cTnT), a highly specific and sensitive biomarker of myocardial injury, has been reported to be a prognostic marker at presentation. But the relationship between serial measurements of serum cTnT and clinical course of the disease has not been described. An illustrative case was a 55-year-old man with normal renal function presenting with congestive heart failure (CHF), whose initial concentration of cTnT was 0.72 ng/ml. Eight samples of initial endomyocardial biopsy specimens showed the presence of myocyte degeneration and interstitial fibrosis with a small amount of amyloid infiltration. However, the cTnT values remained at 0.69 ng/ml after successful management of CHF, and four months later, a second endomyocardial biopsy revealed diffuse massive amyloid protein deposition. He died of CHF, 9 months after initial presentation. In addition, we present 4 cases of amyloidosis with increased serum cTnT levels. We therefore propose that serial measurements of serum cTnT might be helpful for early diagnosis and prediction of prognosis of patients with amyloidosis.

Aged↗
PubMed1996

Performance characteristics of a dialysis-related amyloidosis questionnaire.

To evaluate the effects of beta-2 microglobulin amyloidosis on functional status, a 19-item self-administered questionnaire exploring two major domains-symptoms and disability-was developed as part of this study. Fifteen patients with dialysis-related amyloidosis (DRA) were identified and compared with 15 age-matched control subjects who had been on hemodialysis for less than 24 months. Demographic data, Charlson comorbidity scores, and other clinical and laboratory variables were recorded. Total dialysis-related amyloidosis questionnaire (DRAQ) score (52.1 +/- 16.3 versus 24.4 +/- 6.2, P < 0.0001) and the scores of the symptom (24.5 +/- 7.0 versus 11.5 +/- 2.3, P < 0.0001) and disability (27.5 +/- 10.8 versus 12.9 +/- 4.0, P < 0.0001) subscales were markedly increased among patients with DRA. Baseline characteristics among case and control subjects were similar, except for serum creatinine concentration, which tended to be lower among patients with DRA (8.9 +/- 2.6 versus 11.5 +/- 2.3 mg/dL, P = 0.07). Instrument reliability and internal consistency were high. With a total DRAQ score of 30 or more, the sensitivity and specificity of the instrument were 93% and 80%, respectively. The DRAQ is a reliable, internally consistent, and valid instrument that may be suitable for population screening and clinical practice.

Activities of Daily Living↗
PubMed1995

Secondary amyloidosis associated with Castleman's disease.

A rare case of secondary amyloidosis associated with Castleman's disease is reported. A 53-year-old woman was referred for investigation of proteinuria. Biopsy specimens from kidney and gastric mucosa revealed numerous amyloid deposits, defined as AA amyloidosis by immunohistological staining. Castleman's disease was found in the abdomen as the primary disease for the amyloidosis. Although the urinary protein was somewhat reduced and the inflammatory findings were improved after removal of the lymphoma, renal insufficiency progressed and hemodialysis was begun.

Amyloidosis↗
PubMed2002

A long-survival case of systemic AL amyloidosis with nephrotic syndrome.

A 49-year-old Japanese female was initially diagnosed as having monoclonal gammopathy of undetermined significance in June 1993 (IgG lambda: 3,120 mg/dl). Four years later, she developed AL amyloidosis complicated by nephrotic syndrome and renal failure. Before receiving 5 courses of MP therapy (melphalan plus prednisolone), her serum IgG level had decreased in accordance with the appearance of nephrotic syndrome, which led to the leakage of serum immunoglobulin into the urine. After the discontinuation of the MP therapy, hypogammaglobulinemia has been kept over 24 months, though she still shows a leakage of 4-5 g/day of serum protein, including IgG into the urine. There were no signs of the progression of amyloidosis or renal failure, resulting in a good clinical performance status. Hypogammaglobulinemia due to nephrotic syndrome may have prevented the progression of AL amyloidosis in this case.

Amyloidosis↗
PubMed2002

Long-term follow-up of systemic reactive AA amyloidosis secondary to rheumatoid arthritis: successful treatment with intermediate-dose corticosteroid.

Reactive AA amyloidosis frequently develops in patients with a long history of rheumatoid arthritis (RA), usually resulting in an unfavorable outcome due to dysfunction of the vital organs. We report a 56-year-old woman with this form of amyloidosis secondary to RA who has been successfully treated with intermediate-dose prednisolone for two and a half years since diagnosis. Because prednisolone is superior to other drugs used for amyloidosis with respect to possible harmful effects on vital organs, we can safely try this drug as an alternative therapy to improve the prognosis even in patients with severe organ dysfunction.

Amyloidosis↗
PubMed2003

Nephrotic syndrome due to primary AL amyloidosis, successfully treated with VAD and subsequent high-dose melphalan followed by autologous peripheral blood stem cell transplantation.

Primary AL amyloidosis involves vital organs from the early phase of illness, resulting in a poor prognosis. We report a patient with nephrotic syndrome due to this type of amyloidosis, who was successfully treated with two courses of VAD (vincristine, doxorubicin and dexamethasone) and subsequent high-dose melphalan (140 mg/m2) with autologous stem cell support. Following the serial chemotherapy his proteinuria improved, and M protein became undetectable in both serum and urine. To avoid the progression of primary AL amyloidosis, intensive chemotherapy should be actively used when the general status and vital organ functions are well preserved.

Amyloid↗
PubMed2003

Fatal acute pancreatitis with cystic formation in reactive systemic AA amyloidosis secondary to rheumatoid arthritis.

We report a patient with reactive systemic AA amyloidosis secondary to rheumatoid arthritis who showed fatal acute pancreatitis with a cystic formation in the pancreas head. The pancreatitis rapidly worsened despite intensive treatment and resulted in death. In this patient severe deposition of amyloid in the gastrointestinal tract was considered to play an important role in the pathogenesis of the acute pancreatitis. This is an unusual complication in patients with AA amyloidosis, but we should consider it as a possible diagnosis when patients with AA amyloidosis show recurrent or intractable pain in the upper abdomen.

Acute Disease↗