Duration of treatmen t. Mean age steroids started, y (SD) Dropou ts greater than 20% 8.5 (3.8) No Patients not taking medicatio n

Transcription

1 Refere Medicaio e23 III 0.9 Type udy Propeciv e cohor. paie. corol Duraio reame y (5-8) Mea ared, greaer ha or 8.5 (3.8) Paie o akig Oucome Improveme i he rae developme colioi, lo pulmoary fucio, ad a lo ambulaio reame eve Caarac, weigh gai, re fracure, decreaed boe deiy I er (if wha Reao for ch i er e27 III 2 mg/kg aleraig day e14 III day o/10 day f ad oher udefied doig daily ad aleraeday predioe RCT 28 paie radomized i a 2:1 cheme, bu how may paie were i each group i o clear Predio e: = 16; : = 1 2 y 8.0 (1.3) Plabo Improved fucioal moor core, a lo ambulaio, ad regh reame 117 Duraio reame o defied for all; whe defied varied from 3- o 264-mo equivale 4 year ad older, o oherwie pecified Paie o akig Age a becomig wheelchair boud, requirig par- ad full-ime oivaive veilaio, ad developig colioi by 19 year improve reame Weigh gai, behavioral ch Verebral fracure > dropou, o ieio o rea, o allocaio coalme

2 Refere Medicaio e29 III 0.35 prediolo e e30 III 0.75 predioe e33 III Coricoer oid regime o pecified Type udy RCT, croover. paie. corol 37 DMD, 4 BMD Predio e: = 18; : = 12 Duraio reame 6 mo o reame ad 6 mo o plabo 19 Predio e: 5.49 y ± 1.98; deflazaco r: 5.85 y ± Mea 4.1 (3.4) y Mea ared, 7.8 (2.1); r Predio e: 6.9 (1.0); deflazaco r: 7.45 (0.97) greaer ha or Oucome Plabo Improved fucioal moor core ad regh reame Paie o akig 7.4 (2.5) Paie o akig Improved fucioal moor core, regh, FVC, ad requireme urgery for colioi reame, bu o differe bewee predioe ad Delayed oe cardiomyop ahy (fracioal horeig) i group; a aio ambulaio correlaed duraio eroid ue eve Weigh gai Behavioral ch, caarac, exive weigh gai, hyperei o, ad verebral fracure repored I er (if wha Reao for ch i er primary oucome ideified, o ieio o rea or allocaio coalme

3 Refere Medicaio e18 III 0.75 predioe for he fir 10 day each moh Type udy RCT, croover. paie. corol Duraio reame 17 6 mo reame or plabo, 2 mo wahou, 6 mo reame or plabo Mea ared, 6.29 (0.92) greaer ha or Oucome Plabo Improved fucioal moor core ad regh reame; o ch i QoL durig reame o plabo eve Irriabiliy, cuhigoid appearac e I er (if wha Reao for ch i er e24 III y ± (2.0) Paie o akig Improved fucioal moor core, a lo ambulaio, regh, pulmoary fucio, ad eed for colioi urgery reame Shor aure, caarac; o differe i weigh gai. Paie were oly o ome poeial coudi g charari ic, o allocaio coalme e25 III 0.9 ; 0.6 for he fir 20 day he moh 0.9 : = 32; 0.6 for he fir 20 day: = ad 19, repeciv ely defied (bu over 4 year) Deflazac or 0.9 : 7.6 (1.6) (r 6 8 y); deflazaco r 0.6 fir 20 day: 6.0 (1.5) Paie o akig Improved fucioal moor core, regh, ad developme colioi reame (o p provided) Caarac, fracure

4 Refere Medicaio Type udy. paie. corol Duraio reame Mea ared, (r 4 8 y) greaer ha or Oucome eve I er (if wha Reao for ch i er e15 III Iiiaed a 0.9 ou furher weigh adjume ad redud for ide effec o a idividual bai e34 III 0.75 predioe e37 III 0.75 predioe RCT Preumabl y 9 paie i each reame group y 7.7 (1.2) Paie o akig 7 hiorical corol y ± mo Deflazac or: 8.6 (r ); predio e: 7.5 (r ) provided Naural hiory corol Paie o akig Treame ha a poiive impac o urvival i ecod decade, lo ambulaio, ad pulmoary ad cardiac edpoi Predioe ad equally effecive i improvig mucle regh ad fucioal core Sigifica improveme i peak cough flow ad maximum Caarac ad hor aure, bu o differe i weigh gai Hiruim, cuhigoid appearac e, weigh gai (more predio e), caarac repored Paie were o o baelie charari ic exp ad fucioal core, o ieio o rea

5 Refere Medicaio Type udy. paie. corol Duraio reame Mea ared, greaer ha or Oucome expiraory preure eve I er (if wha Reao for ch i er e49 I 0.75 prediolo e; 10 mg/kg predioe o weeked e50 III 1.25 ad 2.5 mg/kg predioe aleraig -day RCT mo R 4 10 y; for he 4 6 y group: weeked doe 5.8 (0.9), daily doe 5.7 (0.7) RCT 1.25 mg/kg: = 31; 2.5 mg/kg: = 67 Hiorical corol 6 mo R 5 15 y All paie o reame Equivalecy i primary regh ad afey oucome Plabo Aleraeday doig had a le durable beefi for regh ad imilar ide effec daily doig Weigh gai, cuhigoid appearac e, behavior ch Behavior ch, weigh gai, cuhigoid appearac e Paie were o o baelie charari ic, o ieio o rea or allocaio coalme e22 II 0.3 ad 0.75 predioe RCT 67 ( = 34 o 0.3 ad = 34 o 0.75 ) 32 6 mo R 5 15 y; predio e 0.75 : 9.36 (2.86); predio e (2.53) Plabo Improved mucle regh reame, a doe repoe Weigh gai, cuhigoid appearac e, hiruim allocaio coalme

6 Refere Medicaio e31 III 0.3 ad 0.75 predioe ; azahiopri e added laer i udy e38 III Coricoer oid regime o pecified e35 III 0.75 prediolo e for 10 day each moh e45 III 0.75 predioe Type udy RCT Propeciv e cohor. paie = 33 for 0.3 ; = 34 for o, 48 pa eroid ue Predio e: = 36; : = 25; boh: = 14. corol = 33 for 0.3 ; = 34 for o o Duraio reame 6 mo o ad he 12 mo o ad azahiopri e pecified Media 1 y (r 2 mo 9 y) y (r y) Mea ared, R 5 15 y R 4 28 y greaer ha or Oucome Plabo Sigifica improveme i regh ad fucioal core Paie o akig 9.53 (1.2) Paie o akig provided Paie o akig Slower declie i moor ad pulmoary fucio ad developme colioi i eroid group Poiive relaiohip bewee duraio reame prediolo e ad he a oe colioi bu o he everiy colioi a he 17 Improveme i lo ambulaio, mea degree colioi, ad he umber paie eve Weigh gai, cuhigoid appearac e, icreaed blood preure, hor aure Fracure o relaed o eroid ue repored Verebral ad log boe fracure. icreaed weigh I er (if wha Reao for ch i er Paie were o o baelie charari ic, o allocaio coalme

7 Refere Medicaio Type udy. paie. corol Duraio reame Mea ared, greaer ha or Oucome a colioi >10 eve I er (if wha Reao for ch i er e16 III 0.9 ay e43 III Udefied doig predioe or e44 III 0.75 predioe 48 (29 o predio e, 19 o ) Predio e: = 9; : = Mea 15.5 y ( group) ad 14.9 y (orea ed group) 63 3 y ± 2.5 provided 8.5 (3.8) Paie o akig Paie o akig 23 >6 mo 7.5 (0.7) Paie o akig Scolioi >20 ee i group ad 92% o group; moraliy higher i o group (21% v 3%) Lower odd raio developig a abormal horeig fracio reame; o differe bewee predioe ad Le likely o develop vericular dyfucio reame Caarac ad hor aure ee more i group; weigh ch o igifica ed bu o pecified Shorer aure

8 Refere Medicaio e42 III 0.75 predioe for he fir 10 day he moh ha wa wiched o 0.9 a quickly a poible e20 II 0.75 ad 1.5 predioe e27 III 1 Type udy Propeciv e cohor. paie. corol Duraio reame Mea ared, y Media 7 y RCT mo Predio e 0.75 : 9.16 (2.95); predio e 1.5 : 9.09 (2.59) RCT mo R 5 11 y greaer ha or Paie o akig Oucome Improved T2 relaxaio ime ad lef vericular yolic fucio Plabo Improved fucioal moor core, regh, ad FVC o igifica differe bewee he reame group Plabo Improved fucioal moor core ad regh reame eve repored Weigh gai, cuhigoid appearac e, ad hiruim; o differe bewee he 2 regime Cuhigoi d appearac e, icreaed appeie, hiruim, ad behavior ch I er (if wha Reao for ch i er allocaio coalme or ieio o rea (alhough dropou <, o doe o dowgrade furher) Paie were o o baelie charari ic exp weigh ad creaie kiae, o allocaio coalme

9 Refere Medicaio e28 III 0.75 prediolo e e46 III 0.75 predioe e47 III Daily prediolo e; iermie prediolo e (o 10 d/f 10 d); aleraeday prediolo e; daily ; wicher Type udy. paie. corol Duraio reame RCT defied, bu >2 y Propeciv e cohor Propeciv e cohor Daily prediolo e: = 136; iermie prediolo e: = 154; aleraeday prediolo e: = 15; daily : =19; wicher: = 72 Mea ared, 8.83 (1.25) mo R 5 y o lo ambulai o y (r ); 3.6 y (r ); 5.0 y (r ); 4.4 y (r ); 4.1 y (r ), repeciv ely R y greaer ha or Paie o akig All paie o reame Paie o daily v iermie regime Oucome Improved fucioal moor core, lo ambulaio, regh, ad pulmoary fucio reame igifica differe i impac o progreio weake bewee he 2 reame Age a lo ambulaio earlier i iermie group (HR 1.57, CI ); faer declie moor fucio cale i iermie group; o differe i repiraory ad pulmoary fucio eve Weigh gai, cuhigoid appearac e, ifecio Weigh gai; o differe bewee predio e ad More ide effec daily regime: cuhigoid, behavior, hyperei o, hor aure I er (if wha Reao for ch i er Paie were o o baelie charari ic, o allocaio coalme

10 Refere Medicaio Type udy. paie. corol Duraio reame Mea ared, greaer ha or Oucome eve I er (if wha Reao for ch i er e17 III Deflazacor 0.9 or predioe , eiher ACE ihibior or ARB, ad calcium ad viami D = 63 o = 23 o 11.0 y (4.8) 8.6 (3.5)? Paie o o Le deah i eroid group (7/63 v 10/23, p =0.001); urvival rae a 15 y greaer i group (78.6% v 27.9%, p = 0.005); moraliy HR for 0.24 ( ); le cardiomyop ahy i group (7/63 v 14/23, p = ); cardiomyop ahy HR for 0.38 ( ); lower rae declie i LVEF i group (- 0.43% v %, p = 0.01); lower rae declie i FS for Shor aure i eroid group more freque (p < ); o differe i weigh or hyperei o

11 Refere Medicaio Type udy. paie. corol Duraio reame Mea ared, greaer ha or Oucome group (- 0.32% v %, p = 0.002) eve I er (if wha Reao for ch i er e39 III ; 4 paie had ake predio e previouly 13; ad addiioa l hiorical corol Year, oherwie o defied for all paie; follow-up r mo provided Naural hiory corol a well a paie o o reame Improved fucioal moor core, regh, ad pulmoary fucio reame; o ch i cardiac oucome Shor aure, caarac, obeiy, fracure While paie were mached for baelie, here i o quaiaive compario, ad here i o compario oher baelie charari ic, o allocaio coalme, o ie o rea, ad o allocaio

12 Refere Medicaio Type udy. paie. corol Duraio reame Mea ared, greaer ha or Oucome eve I er (if wha Reao for ch i er coalme e48 III 0.75 mg/kg predioe 10 d o/10 d f; 0.75 mg/kg predioe 10 d o/20 d f; 0.75 predioe 30;1;1, repecivel y; = 33 o predio e 14 Mea follow-up 57 mo bu legh reame o pecified Media 5 y (r ) Paie o akig differe i heigh ad weigh bewee he 4 ad 9 y bewee paie ad corol repored e36 III 0.75 mg/kg aleraig -day prediolo e Propeciv e cohor y (r 1.5 5) provided Paie o akig Improved a lo ambulaio ad rae developig colioi reame repored Abbreviaio: ACE = agioei-coverig ezyme; ARB = agioei repor blocker; BMD = Becker mucular dyrophy; CI = cide ierval; DMD = Duchee mucular dyrophy; FS = fracioal horeig; FVC = ford vial capaciy; HR = hazard raio; LVEF = lef vericular ejecio fracio; = o applicable; QoL = qualiy life; RCT = radomized corolled rial.