433.2 ๐Ÿ“š ๅœ‹่€ƒ็‰ˆ๏ผˆ้†ซๅธซๅœ‹่€ƒ / PGY OSCE๏ผ‰


433.2.0.1 ๐Ÿ“Œ Cram Sheet

433.2.0.1.1 ๐Ÿ”ฅ ้ซ˜ yield 10
  1. Cystinuria๏ผšSLC3A1 / SLC7A9๏ผ›ๅ…ญ่ง’ๅฝข็ตๆ™ถ๏ผ›ๆฒป็™‚๏ผš> 4 L waterใ€้นผๅŒ–๏ผˆpH > 7.5๏ผ‰ใ€tiopronin
  2. Hartnup disease๏ผšSLC6A19๏ผ›ไธญๆ€ง AA aminoaciduria๏ผ›Pellagra-like + ataxia๏ผ›ๆฒป๏ผšnicotinamide
  3. Cystinosis๏ผšCTNS๏ผ›lysosomal cystine ็ดฏ็ฉ๏ผ›Fanconi + ๅคš้‡ๅ…งๅˆ†ๆณŒ็ผบ้™ท๏ผ›ๆฒป๏ผšcysteamine
  4. LPI๏ผšSLC7A7๏ผ›dibasic AA basolateral๏ผ›้ซ˜่ก€ๆฐจ๏ผ›ๆฒป๏ผšcitrulline ่ฃœๅ…… + ่›‹็™ฝ้™ๅˆถ
  5. Citrin deficiency (CTLN2)๏ผšSLC25A13๏ผ›ไบžๆดฒๅธธ่ฆ‹๏ผ›ๅ่‚‰้ฃŸใ€้ฟ็ณ–๏ผ›ๆ€ฅๆ€ง้ซ˜่ก€ๆฐจ๏ผ›ๆฒป๏ผš้™็ณ–ใ€่‚็งปๆค
  6. Cystinuria ็—…ไบบ 24h urine cystine 2400-7200 ฮผmol/d๏ผˆๆญฃๅธธ < 250๏ผ‰
  7. Cystine ๅœจ urine pH < 7.5 ไธๆ˜“ๆบถ๏ผ›> 7.5 ๆบถ่งฃๅบฆๅคงๅขž
  8. Hartnup ไธ‰ D๏ผšDermatitisใ€Diarrheaใ€Dementia/Delirium
  9. Cystinosis ไธ‰ๅž‹๏ผšclassic nephropathic๏ผˆๆœ€ๅšด้‡๏ผ‰ใ€juvenile nephropathicใ€ocular non-nephropathic
  10. Citrin deficiency ่ˆ‡ๅ…ถไป–ๅฐฟ็ด ๅพช็’ฐ็–พ็—…็š„ๅทฎๅˆฅ๏ผšglutamine ๆญฃๅธธ๏ผˆๅ…ถไป– โ†‘๏ผ‰๏ผ›galactose-1-P โ†‘
433.2.0.1.2 ๐Ÿ”ข ๅฟ…่ƒŒๆ•ธๅญ—
้ …็›ฎ ๆ•ธๅญ—
Cystinuria ็››่กŒ็އ 1/7000
่…Ž็ต็Ÿณไธญ cystine ๅ ๆฏ” 1-2% (ๆˆไบบ); 4-5% (ๅ…’็ซฅ)
Cystine ้ฃฒๆฐด้‡ > 4 L/d๏ผˆ็†ๆƒณ 5-7 L๏ผ‰
็›ฎๆจ™ urine cystine < 1000 ฮผmol/L (250 mg/L)
็›ฎๆจ™ urine pH > 7.5
Hartnup ็››่กŒ็އ 1/24,000
Hartnup nicotinamide 50-250 mg/d
Cystinosis ็››่กŒ็އ 1/100,000-200,000
Cystinosis ESRD without tx 10 ๆญฒๅ‰
Cysteamine ็ถญๆŒๅŠ‘้‡ 1.3 g/mยฒ /d ๅˆ† 2 ๆฌก
LPI Finland ็››่กŒ็އ 1/60,000
LPI citrulline ๅŠ‘้‡ 2-8 g/d
CTLN2 ไบžๆดฒ็››่กŒ็އ ~ 1/20,000

433.2.0.2 โญ ้ซ˜ yield

433.2.0.2.1 ไธป่ฆ transport defect ่กจ
็—… SLC ๅŸบๅ›  Substrate ่กจ็พ ๆฒป็™‚
Cystinuria SLC3A1, SLC7A9 Cystine + lysine + arg + ornithine ๅ่ฆ†่…Ž็ต็Ÿณ ๅ–ๆฐดใ€้นผๅŒ–ใ€tiopronin
Lysinuric protein intolerance SLC7A7 Dibasic AA (basolateral) ้ซ˜่ก€ๆฐจใ€osteoporosisใ€PAP Citrullineใ€่›‹็™ฝ้™ๅˆถ
Hartnup SLC6A19 Neutral AA Pellagra-like + ataxia Nicotinamide
Cystinosis CTNS Lysosomal cystine Fanconiใ€ๅคš้‡ๅ…งๅˆ†ๆณŒ Cysteamine
Citrullinemia type 2 SLC25A13 Mito aspartate-glutamate ้ซ˜่ก€ๆฐจใ€ๅ่‚‰้ฃŸ ้™็ณ–ใ€่‚็งปๆค
HHH syndrome SLC25A15 Mito ornithine ้ซ˜่ก€ๆฐจใ€homocitrullinuria ้™่›‹็™ฝ + citrulline
433.2.0.2.2 Cystinuria ๆฒป็™‚้šŽๆขฏ
  1. Fluid > 4 L/d๏ผˆๅŠๅคœ่ตทไพ†ๅ–๏ผ‰
  2. ้นผๅŒ–๏ผˆpotassium citrate๏ผ‰pH > 7.5
  3. ๆธ›้ˆ‰ + ๆธ›ๅ‹•็‰ฉๆ€ง่›‹็™ฝ
  4. Tiopronin 800-1200 mg/d ๆˆ– penicillamine 1-3 g/d
  5. ็ต็Ÿณ็งป้™ค๏ผšESWL / URS / PCNL
  6. ๆœซๆœŸ๏ผš่…Ž็งปๆค
433.2.0.2.3 Hartnup ไธ‰ D
  • Dermatitis (ๅ…‰ๆšดๆ›ฌ้ƒจไฝ)
  • Diarrhea
  • Dementia / Delirium / cerebellar ataxia
433.2.0.2.4 Cystinosis ๅคš้‡ๅ…งๅˆ†ๆณŒ็ผบ้™ท๏ผˆๅ…งๅˆ†ๆณŒ็ง‘่ฆ่จ˜๏ผ๏ผ‰
  • ็”ฒ็‹€่…บไฝŽไธ‹ โ†’ levothyroxine
  • ็ณ–ๅฐฟ็—… โ†’ insulin
  • ๆ€ง่…บไฝŽไธ‹ (primary, male) โ†’ testosterone
  • ็”Ÿ้•ท้ฒ็ทฉ โ†’ growth hormone
  • ้’ๆ˜ฅๆœŸๅปถ้ฒ
  • Hypohydrosis๏ผˆ็„กๆฑ—๏ผ‰
433.2.0.2.5 Bartter vs Gitelman ้€Ÿ่จ˜
้ …็›ฎ Bartter Gitelman
็ผบ้™ทไฝ็ฝฎ TAL DCT
ๅฐๆ‡‰ๅˆฉๅฐฟๅŠ‘ Loop (furosemide) Thiazide
24h urine Ca โ†‘ โ†“
24h urine Mg ๆญฃๅธธๆˆ– โ†“ โ†‘
่ก€ Mg ๅคšๆญฃๅธธ โ†“
่กจ็พๅนด้ฝก ๅฌฐๅ…’/ๅ…’็ซฅ ๆˆไบบ
่ก€ๅฃ“ ๆญฃๅธธๆˆ– โ†“ ๆญฃๅธธ

433.2.0.3 ๐ŸŽฏ ่‡ชๆˆ‘ๆชขๆธฌ

  1. Cystinuria ็š„ๅ››ๅ€‹ๅฐฟ้Ž้‡ AA๏ผŸ โ†’ Cystine, lysine, arginine, ornithine
  2. Cystinuria ็ต็Ÿณ็š„ unique ็ตๆ™ถๅฝข็‹€๏ผŸ โ†’ ๅ…ญ่ง’ๅฝข
  3. Cystine ๆบถ่งฃๅบฆ็ชๅขž็š„ pH๏ผŸ โ†’ > 7.5
  4. Hartnup ไธ‰ D๏ผŸ โ†’ Dermatitis, diarrhea, dementia/delirium
  5. Hartnup ่ฃœๅ……ไป€้บผ็ถญไป–ๅ‘ฝ๏ผŸ โ†’ Nicotinamide
  6. Cystinosis ๆฒป็™‚่—ฅ๏ผŸ โ†’ Cysteamine bitartrate
  7. Cystinosis ๆœ€ๅšด้‡ๅž‹็š„่…Ž่‡Ÿ็—‡็‹€๏ผŸ โ†’ Renal Fanconi syndrome
  8. Cystinosis ็‚บไป€้บผๅ…งๅˆ†ๆณŒ็ง‘่ฆ่ฟฝ๏ผŸ โ†’ ๅคš้‡ๅ…งๅˆ†ๆณŒ็ผบ้™ท๏ผˆ็”ฒ็‹€่…บใ€DMใ€ๆ€ง่…บใ€็”Ÿ้•ท๏ผ‰
  9. LPI ๆ€ฅๆ€ง็™ผไฝœ็š„็‰นๅพตๅฏฆ้ฉ—ๅ€ผ๏ผŸ โ†’ ้ซ˜่ก€ๆฐจ + ่›‹็™ฝไธ่€
  10. LPI ๆฒป็™‚้—œ้ต่—ฅ๏ผŸ โ†’ Citrulline + ่›‹็™ฝ้™ๅˆถ
  11. Citrin deficiency ้ฃฒ้ฃŸ็‰นๅพต๏ผŸ โ†’ ่‡ช็™ผ้ฟ็ณ–ใ€ๅ่‚‰้ฃŸ
  12. Citrin deficiency ่ˆ‡ๅ…ถไป–ๅฐฟ็ด ๅพช็’ฐ็–พ็—…้‘‘ๅˆฅ้‡้ปž๏ผŸ โ†’ Glutamine ๆญฃๅธธ + galactose-1-P โ†‘
  13. Bartter vs Gitelman 24h urine Ca๏ผŸ โ†’ โ†‘ vs โ†“
  14. Gitelman ๅฐๆ‡‰ๅ“ช็จฎๅˆฉๅฐฟๅŠ‘๏ผŸ โ†’ Thiazide (SLC12A3 = NCC)
  15. ใ€Œๅคฉ็„ถ furosemide ไธญๆฏ’ใ€ๆ˜ฏๅ“ชๅ€‹็—…๏ผŸ โ†’ Bartter

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