Therapeutic options in systemic AL amyloidosis.

Systemic amyloid light chain (AL) amyloidosis is a severe disease with unfavourable prognosis. Since the late 1970s different therapeutic modalities in AL amyloidosis have been investigated, trying to prolong survival. This review deals with the therapeutic modalities in AL amyloidosis to date, and highlights future perspectives.

Diagnostic and therapeutic approach of systemic amyloidosis.

Amyloidosis is a group of diseases, all characterised by deposition of protein fibrils with a beta-sheet structure. This structure generates affinity of amyloid for Congo red dye and is resistant to proteolysis. Three types of systemic amyloidosis are important for the clinician: AA (related to underlying chronic inflammation), AL (related to underlying monoclonal light chain production) and ATTR amyloidosis (related to old age or underlying hereditary mutations of transthyretin). Signs and symptoms vary considerably among the three types and the choice of treatment differs completely. A stepwise approach in diagnosis and therapy is presented. When amyloidosis is suspected the first step is histological proof of amyloid and the second is proof of systemic involvement. The next two steps are determination of the type of amyloid followed by detection of the precursor protein. The fifth step is a thoughtful clinical evaluation, necessary for assessment of prognosis and therapy. Subsequently, the choice of therapy is based on the 'precursor-product' concept. In the final step, the effects of therapy on the underlying disease as well as on the amyloidosis are assessed during follow-up. In this evaluation serum amyloid P component (SAP) scintigraphy helps to show organ involvement and therapy response.

Pyridoxin-responsive anaemia in a patient with a history of polycythaemia vera.

Pancytopenia in the course of polycythaemia vera (PV) following the proliferative and stable phase, ultimately leads to a spent phase characterized by extensive marrow fibrosis. We describe a patient with a history of PV and pancytopenia caused by myelodysplasia, before a genuine end stage myelofibrosis had occurred. The related anaemia was responsive to pyridoxin.

Cystitis and interstitial nephritis related to the use of tiaprofenic acid (Surgam).

We describe a case of concurrent presentation of severe haemorrhagic cystitis and acute interstitial nephritis with eventually lethal outcome, associated with the use of tiaprofenic acid (Surgam), a propionic acid-derived non-steroidal anti-inflammatory drug (NSAID). Although interstitial nephritis and haemorrhagic cystitis are caused by all types of NSAIDs, a higher incidence of interstitial nephritis and especially haemorrhagic cystitis has been described related to propionic acid derivatives. Awareness of this serious adverse effect is important.