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PORPHYRIASDefinition: They are inherited or acquired defects of enzymes in the heme biosynthetic pathway. Steroid hormones, drugs andnutrition influence the production of porphyrin precursors and porphyrins thereby precipitating or increasing the severity of someporphyrias. Therefore porphyrias are multifactorial.Classification: They are classified as HEPATIC AND ERYTHROPOETIC DEPENDING ON THE PRIMARY SITE OF OVERPRODUCTION ANDACCUMULATION OF PORPHYRINOGEN/PORPHYRIN.HEPATIC PORPHYRIAS:
Acute intermittent porphyria (acute)
Variegate porphyria (acute)
Hereditary coproporphyria (acute)
ALA dehydratase deficiency
Porphyria cutanea tarda.
Hepatoerythropoetic porphyriaERYTHROPOETIC PORPHYRIA:
Erythropoetic protoporphyria
Congenital erythropoetic porphyriaWhat are porphyrins?They are cyclic molecules formed by the linkage of 4 pyrrole rings through methynyl bridges. Different porphyrins vary in thenature of side chains attached to each of the 4 pyrrole rings. For eg. uroporphyrin contains acetate and propionate side chain.Coproporphyrin contains methyl and propionate side chains. The side chains are arranged around the nucleus in 4 different waysdesignated by roman numerals --- I –IV. Only type III which contains an asymmetric distribution of ring D are physiologically importantin humans. Porphyrin precursors exist in the chemically reduced form called porphyrinogens. In contrast to porphyrins which arecoloured, the porphyrinogens are colorless. Porphyrinogens serve as intermediates between porphobilinogen and protoporphyrin inHEME biosynthesis.Porphyrins are cyclic compounds that readily bind metal ions usually ferrous or ferric ions. The most prevalentmetalloporphyrin is heme. Heme is essential for ----
Oxygen binding and transport (as hemoglobin and myoglobin)
For electron transport (cytochromes) and for
Mixed function oxidases such as cytochrome P450.PATHOPHYSIOLOGY OF CUTANEOUS LESIONS IN PORPHYRIA --------Porphyrins have unique photo biologic and spectroscopic properties. Metal free porphyrins e.g. URO, COPRO, PROTO in acidicsolutions show a major absorption peak in 400-410 nm region (Soret band), they also exhibit 4 additional absorption bands withdecreasing intensity between 500-700 nm. This results in 2 major fluorescence emission peaks at 600-610 nm and 640-660 nmregions. There is no single clearly defined pathway that can explain photosensitization evoked by porphyrins and light.Porphyrin -----------------------
Excited porphyrins (singlet and triplet state)↓highly reactive+ OxygenReactive oxygen speciesLipid peroxidesDamaged membranes↓consequencesMast cell degranulationHemolysisTissue damageFACTORS CONTIBUTING TO THE DEVELOPMENT OF CUTANEOUS LESIONS IN PORPHYRIA:
Sunlight (380-760 nm) : blue and red light
Reactive oxygen species
Cells ---- erythrocyes, mast cells, PMNs, fibroblasts
Soluble mediators ---- complement system, Factor XII dependant pathways, Eicosanoids, matrix metalloproteinases.HEME BIOSYNTHETIC PATHWAY AND DIFFERENT PORPHYRIAS:STEP 1:Succinyl CoA + Glycine = ALAEnzyme = ALA synthase -------- a rate limiting enzyme. Heme has an inhibitory action on this enzyme. The activity of this enzyme is increased in those hepatic porphyrias in which acute attacks characterized by a neurologic visceralsymptom complex occur as in AIP, VP, HCP.STEP 2:2 X ALA = PorphobilinogenEnzyme = ALA dehydratasePorphyria = ALA dehydratase deficiency (HEPATIC PORPHYRIA)C/F: like AIP.STEP 3:4 X PBG = Hydroxymethybilane which cyclizes spontaneously to form UROPOPHYRINOGEN 1.Enzyme = PBG deaminasePorphyria = acute intermittent porphyria (HEPATIC PORPHYRIA).
 
Autosomal dominant condition. The disorder is latent in majority and usually precipitated after puberty by DRUGS, HORMONALFACTORS, ALCOHOL, FASTING, SURERY, MENSTRUAL CYCLE OR PREGNANCY.C/F: It is characterized by acute attacks of GI and neuropsychiatric symptoms. No cutaneous lesions. THE URINE IS PORTWINECOLORED.Investigations:
Urinary excretion of ALA and PBG are elevated during and between the attacks.
2 rapid screening tests are available to test freshly voided urine for increased PBG. The first is simply to expose the urine tobright sunlight for several hours ------- darkening to a deep red colour suggests that PBG is present. The second test knownas the HOESCH TEST is also a simple procedure to detect the urinary PBG. To 2 ml of Ehrlich’s reagent, 2 drops of freshlyvoided urine is added, a cherry red color of the sample gives a positive result.STEP 4:Hydroxymethybilane ------------- uroporphyrinogen IIIEnzyme = UROGEN III Synthase.Porphyria = CONGENITAL ERYHTROPOETIC PROPHYRIA (CEP).AUTOSOMAL RECESSIVEIt results in the accumulation of hydroxymethybilane which converts spontaneously to UROGEN I.Onset = in the first few months of life.C/F: There is moderate to severe photosensitivity. CEP CAUSES THE MOST MUTILATING SKIN LESIONS OUT OF ANY OF THEPORPHYRIAS. The photosensitivity is due to the excess URO I and COPRO I in the RBCs, plasma and skin which may cause photolysisof porphyrin rich cells. The skin manifestations include skin fragility and vesicles and bulla which may contain pink fluorescent fluid.Secondary infection, delayed healing and scar formation may occur. This may lead to loss of acral tissue such as tips of ears, nose,fingers and facial mutilation. Other features are ----
Hirsutism of the photoexposed skin
Irregular hypo and hyperpigmentation
Cicatricial alopecia
Eye changes ---- photophobia, keratoconjunctivitis, ectropion, symblepheron
Erythrodontia
Urine ---- fluoresce pink
Splenomegaly
hypersplenism
Hemolytic anemia
Porphyrin rich gallstone.INVESTIGATIONS:
Increased excretion of URO I and COPRO I in urine ------ appears burgundy red.
Increased COPRO I in the feces.
Marrow normoblasts exhibit relatively stable fluorescence and contain markedly elevated URO I, COPRO I and PROTO.STEP 5:UROGEN III -------------- COPROGEN IIIEnzyme = UROGEN decarboxylase.Porphyria = Porphyria cutanea tardaIt is the most common form of porphyria. The disease is classified into 2 forms --------
 Type I ---- acquired/sporadic. The enzyme is deficient only in the liver. The disease manifests usually by 4
th
to 5
th
decade when the subject is exposed to some environmental agents like alcohol, estrogens, iron, viral infections(HCV, HIV), polychlorinated hydrocarbons particularly HCB, dioxin(environmental pollutant).
 Type II ---- hereditary PCT. It is inherited as AD. The enzyme is reduced to 50% in all tissues including RBCs andcultured skin fibroblasts. Penetrance is very low. It may manifest in childhood.C/F:
 The most common skin manifestation of PCT is SKIN FRAGILITY manifesting as erosions, vescicles and bulla in thephotoexposed and trauma prone areas --------- usually on the dorsa of hands and feet. They resolve with hypo andhyperpigmented scarring and milia.
In contrast to EPP, the subjective discomfort following sun exposure is distinctly uncommon in PCT. In fact many patients donot realize that their skin eruptions are causally related to sunlight.
Other cutaneous changes include facial hypertrichosis along the temples and cheeks, mottled pigmentation resemblingchloasma. There may be purplish red suffusion of the central part of the face particularly involving the periorbital areas.Sclerodermoid plaques can occur (both in PCT and HEP). They occur both in photoexposed and photo covered areas.Histopathology:In mild cases, homogenous Eosinophilic deposits are limited to the immediate vicinity of papillary dermal vessels best seen inPAS. In severe cases, these further extend and coalesce with each other. In addition the deeper vessels may show homogenousmaterial in the walls and around the eccrine glands.Drugs that are unsafe in acute porphyria:
1.
ANTICONVULSANTS---- barbiturates, valproate, trimethadione, ethosuximide, hydantoin, carbamazepine
2.
OTHER CNS DRUGS---- hydantoin, nitrazepam, diazepam, nortryptiline, clordiazepoxide, fentanyl.
3.
ANTIBIOTICS----- chloramphenicol, sulfonylurea, dapsone, Griseofulvin, chloroquin, erythromycin, nalidixic acid,rifampicin, INH, nitrofurantoin, pyrizinamide, cephalosporin
4.
HORMONES----- Estrogen, progesterone, chlorpropamide, tolbutamide, Danazol,
5.
PAIN KILLERS---- diclofenac, pentazocine, phenylbutazone,6.HEAVY METAL
 
The bullae arise subepidermally. The dermal papilla on the floor of the bulla extends into the bulla cavity ---------- calledfestooning.The epidermis forming the roof often contains Eosinophilic bodies that are elongated and sometimes segmented called“caterpillar bodies”. They are PAS + and diastase resistant. These bodies contain colloid, cellular organelles and electron densematerial thought to be of BM origin.Lab findings:
 The total body iron is raised.
Urine shows elevated levels of uroporphyrins (isomers I > isomers III). And also 7-, 6-, 5- and 4- carboxyl porphyrins. Thesefindings account for the orage red fluorescence of the urine under Wood’s lamp.
In stool, there is increased excretion of ISOCOPROPORPHYRINOGEN with lesser amounts of uroporphyrin, coproporphyrinand 7- carboxyl porphyrin.
Screening tests ----- acidy a random urine sample with a few drops of 10% HCl or acetic acid and look for orange redfluorescence.
Complete blood count for Hb and hematocrit levels prior to therapy
Liver enzymes and hepatitis and HIV virus screening.Treatment:1.Avoid alcohol, estrogens, iron and any drugs that can exacerbate.
2.
Sunscreen mainly containing titanium oxide to absorb the soret band (400 +/- 10 nm).
3.
Phlebotomy is the treatment of choice. 500 ml of blood is removed weekly or biweekly until Hb drops to 10-11. 5-6phlebotomy are needed for remission. Clinical improvement is seen after 3-6 months. Remissions range frommonths to years.
4.
Alternately, antimalarials (HCQS 200 mg twice weekly) may be given. They form water soluble complex withporphyrins.
5.
Less commonly used treatments are --------- cholestyramine, iron chelators, high dose hydroxychloroquin anderythropoietin.STEP 6:COPROGEN III ------------- PROTOPORPHYRINOGEN IXEnzyme = Coproporphrinogen oxidase.Porphyria = Hereditary coproporphyriaADCharacterized by acute attacks similar to AIP and cutaneous lesions resembling PCT.STEP 7:PROTOPORPHYRINOGEN IX -------- PROTOPORPHYRIN IXEnzyme = Protoporphyrinogen oxidasePorphyria = Variegate porphyria.RBC ferrochelatase enzyme is also reduced by 50%.Manifests after pubertyThere are a combination of features of AIP and PCT.Investigations:
Elevated fecal excretion of porphyrins is the main biochemical feature of VP (proto> copro). A rapid screening test is to mixthe feces with equal amounts of alcohol, ether and glacial acetic acid. Any porphyrin present will fluoresce pink under woodslight.
Fecal X porphyrin, a relatively ether insoluble group of heterogenous porphyrin peptide conjugates are raised in VP more sothan in other porphyria.
PBG and ALA are raised only during the attacks not between the attacks unlike AIP where these porphyrins are raised bothduring and between the attacks.
Urinary coproporphyrin is moderately elevated and higher than uroporphyrin. This is in contrast to PCT where uroporphyrin> coproporphyrin.
Plasma of these patients contain a porphyrin with a fluorescence emission maximal at 626 nm that is specific for VP.
VP and PCT may coexist.STEP 8:PROTOPORPHYRIN IX ---------- HemeEnzyme = FerrochelatasePorphyria = Erythropoetic protoporphyria (EPP)C/F:
Starts in childhood ( mean age = 4 years).
 There is photocutaneous changes and liver disease.
Symptoms occur within a few minutes to 1 hour of exposure to sunlight in exposed areas. The activating wavelength are400 nm and to a lesser extent 500-600 nm. Exposure to sunlight through window glass will thus precipitate as also artificiallights.
 They complain of burning and tingling sensation more than itching. This is followed by ERYTHEMA, OEDEMA ANDOCCASIONALLY PURPURA IN THE SUN EXPOSED AREAS.
Chronic changes include pock like scars on nose and cheeks, thickened leathery skin and lichenoid papules over the dorsalhands, nose, cheeks, lips and ears.
Rarely cholelithiasis and liver failure may occur.
BULLA, SKIN FRAGILITY, PIGMENTATION, HYPERTRICHOSIS OR MUTILATION is uncommon in EPP.
Symptoms improve spontaneously after the age of 10-11 years.Investigations:
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