Home-based subcutaneous immunoglobulin G replacement therapy under real-life conditions in children and adults with antibody deficiency


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Subcutaneous immunoglobulin (SCIG) therapy is an alternative to intravenous immunoglobulin (IVIG) therapy. Methods We evaluated the efficacy and safety of the SCIG Vivaglobin ® (formerly known as Beriglobin ® SC) under real-life conditions in a post-marketing observational study in 82 patients with primary or secondary antibody deficiencies. Health-related quality of life (HRQoL) was evaluated in a subset of 30 patients previously treated with IVIG (including 11 children < 14 years) using the Short Form 36 (SF-36) for patients ≥ 14 years of age (adults) and the Child Health Questionnaire - Parental Form 50 (CHQ-PF50) for children < 14 years of age. Treatment preferences were assessed in adults. Results The mean serum immunoglobulin G (IgG) trough level during SCIG treatment (7.5 g/L) was higher than during previous IVIG treatment (6.6 g/L; p < 0.01). The investigators assessed the efficacy of SCIG therapy as "excellent" in 89% of patients. No systemic adverse drug reactions were observed. Improvements by ≥ 5 points were observed in 5 of 8 SF36 subscales and in 6 of 12 CHQ-PF50 subscales. Statistically significant improvements (p ≤ 0.05) were observed for the SF-36 subscales of bodily pain, general health perceptions, and vitality (adults), and for the CHQ-PF50 subscales of general health perceptions, parental impact - time, parental impact - emotional, and family activities (children). Patients preferred SCIG over IVIG therapy (92%) and home therapy over therapy at the clinic/physician (83%). Conclusion This study confirms that therapy with Vivaglobin ® at home is effective, safe, well tolerated, and improves quality of life in patients with antibody deficiency.



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Published 01 January 2010
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238 EUr J MeD ReS (2010) 15: 238-245
EuRoPEan JouRnal of MEdIcal REsEaRcH
JUNe 28, 2010
© I. HOLzàpFeL PUBLiSherS 2010
HoME-basEdsubcutanEousIMMunoglobulIng REPlacEMEnt tHERaPy undERREal-lIfEcondItIons IncHIldREn andadults wItHantIbodydEfIcIEncy
1 22 21 f. HOFFmàNN, b. grimBàCher, J. thieL, H.-H. PeTer, b. H. beLOhràDSkY ® àND The VivàGLOBiNsTUDY grOUp
1 uNiverSiTY chiLDreN’S HOSpiTàL, PàeDiàTriC INFeCTiOUS diSeàSeS àND ImmUNOLOGY, lUDWiG MàximiLiàNS uNiverSiTY, MUNiCh, germàNY, 2 uNiverSiTY HOSpiTàL, ImmUNOLOGY àND RheUmàTOLOGY, ceNTre OF chrONiC ImmUNODeFiCieNCY, uNiverSiTY OF freiBUrG, germàNY
Abstract Backgr ound:sUBCUTàNeOUS immUNOGLOBULiN (scIg) TheràpY iS àN àLTerNàTive TO iNTràveNOUS immUNOGLOBU-LiN (IVIg) TheràpY. Methods:Thewe evàLUàTeD The eFFiCàCY àND SàFeTY OF ® ® scIg VivàGLOBiN(FOrmerLY kNOWN àS beriGLOBiN sc) UNDer reàL-LiFe CONDiTiONS iN à pOST-màrkeTiNG OB-ServàTiONàL STUDY iN 82 pàTieNTS WiTh primàrY Or SeC-ONDàrY àNTiBODY DeFiCieNCieS. HeàLTh-reLàTeD qUàLiTY OF LiFe (HRQOl) WàS evàLUàTeD iN à SUBSeT OF30 pàTieNTS previOUSLY TreàTeD WiTh IVIg (iNCLUDiNG 11 ChiLDreN <14 YeàrS) USiNG The shOrT fOrm 36 (sf-36) FOr pà-TieNTS ≥ 14 YeàrS OFàGe (àDULTS) àND The chiLD HeàLTh QUeSTiONNàire - PàreNTàL fOrm 50 (cHQ-Pf50) FOr ChiLDreN <14 YeàrS OFàGe. treàTmeNT preFereNCeS Were àSSeSSeD iN àDULTS. Results:the meàN SerUm immUNOGLOBULiN g (IGg) TrOUGh LeveL DUriNG scIg TreàTmeNT (7.5 G/l) WàS hiGher ThàN DUriNG previOUS IVIg TreàTmeNT (6.6 G/l; p<0.01). the iNveSTiGàTOrS àSSeSSeD The eFFiCàCY OF scIg TheràpY àS “exCeLLeNT” iN 89% OFpàTieNTS. nO SYSTemiC àDverSe DrUG reàCTiONS Were OBServeD. Im-prOvemeNTS BY ≥5 pOiNTS Were OBServeD iN 5 OF8 sf-36 SUBSCàLeS àND iN 6 OF12 cHQ-Pf50 SUBSCàLeS. sTà-TiSTiCàLLY SiGNiFiCàNT imprOvemeNTS (p≤0.05) Were OB-ServeD FOr The sf-36 SUBSCàLeS OFBODiLY pàiN, GeNeràL heàLTh perCepTiONS, àND viTàLiTY (àDULTS), àND FOr The cHQ-Pf50 SUBSCàLeS OFGeNeràL heàLTh perCepTiONS, pàreNTàL impàCT - Time, pàreNTàL impàCT - emOTiONàL, àND FàmiLY àCTiviTieS (ChiLDreN). PàTieNTS preFerreD scIg Over IVIg TheràpY (92%) àND hOme TheràpY Over TheràpY àT The CLiNiC/phYSiCiàN (83%). Conclusion:thiS STUDY CONFirmS ThàT TheràpY WiTh Vi-® vàGLOBiN àThOme iS eFFeCTive, SàFe, WeLL TOLeràTeD, àND imprOveS qUàLiTY OFLiFe iN pàTieNTS WiTh àNTiBODY DeFi-CieNCY.
Abbr eviations:cHQ-Pf50: chiLD HeàLTh QUeSTiON-Nàire – PàreNTàL fOrm 50; HRQOl: heàLTh-reLàTeD qUàL-iTY OFLiFe; IGg: immUNOGLOBULiN g; IVIg: iNTràveNOUS immUNOGLOBULiN; scIg: SUBCUTàNeOUS immUNOGLOBU-LiN; sf-36: HRQOl qUeSTiONNàire shOrT fOrm 36
PrimàrY àNTiBODY DeFiCieNCieS BeLONG TO The WiDe ràNGe OFprimàrY immUNe DeFiCieNCY DiSeàSeS CàUSeD BY iNTriNSiC Or GeNeTiC DeFeCTS iN The immUNe SYSTem [1, 2]. theY COmpriSe à ràNGe OFiNheriTeD DiSOrDerS ThàT àre ChàràCTerizeD BY DeFeCTS iN àNTiBODY prODUC-TiON àND/Or FUNCTiON. cOmmON vàriàBLe immUNODeFi-CieNCY, WhiCh iS The mOST COmmON OFThe mOre Severe primàrY immUNODeFiCieNCieS, iS àSSOCiàTeD WiTh reCUr-reNT Or ChrONiC iNFeCTiONS OFThe reSpiràTOrY TràCT (>95%) àND The GàSTrOiNTeSTiNàL TràCT (àpprOximàTeLY 50%), àS WeLL àS WiTh àUTOimmUNe pheNOmeNà (20% TO 30%), SpLeNOmeGàLY (àpprOximàTeLY 30%), NON-CàSeàTiNG GràNULOmàS (àpprOximàTeLY 10%), àND màLiG-NàNCieS [3, 4]. PàTieNTS SUFFeriNG FrOm primàrY àNTiBODY DeFiCieN-CieS reqUire LiFeLONG reGULàr immUNOGLOBULiN iNFUSiONS [2, 5]. nOWàDàYS, SUBCUTàNeOUS immUNOGLOBULiN (scIg) iNFUSiONS àre USeD àS àN àLTerNàTive TO iNTrà-veNOUS immUNOGLOBULiN (IVIg) àDmiNiSTràTiON iN àDULTS àND ChiLDreN WiTh primàrY Or SeCONDàrY àNTi-BODY DeFiCieNCieS [6, 7]. scIg iNFUSiONS Were ShOWN TO Be eFFiCàCiOUS, SàFe, àND WeLL TOLeràTeD [8]. theY àre àLSO àppreCiàTeD BY àDULTS àND ChiLDreN FOr Their eàSe OF àDmiNiSTràTiON[8, 9]. feW SYSTemiC àDverSe reàC-TiONS hàve BeeN repOrTeD DUriNG scIg iNFUSiONS, iNDi-CàTiNG à FàvOUràBLe SàFeTY prOFiLe COmpàreD TO iNTrà-veNOUS Or iNTràmUSCULàr àDmiNiSTràTiON OFim-mUNOGLOBULiN [10, 11]. the àveràGe SerUm LeveLS àT-TàiNeD DUriNG scIg TheràpY àre COmpàràBLe TO ThOSe àTTàiNeD DUriNG IVIg TheràpY, BUT WiTh reDUCeD OSCiL-LàTiONS BeTWeeN CONSeCUTive iNFUSiONS [9, 12, 13]. HOme-BàSeD scIg TreàTmeNT iS CONveNieNT BeCàUSe FeWer viSiTS TO The CLiNiC Or phYSiCiàN àre reqUireD ThàN WiTh IVIg TheràpY, WhiCh iS NOT LiCeNSeD àS hOme TreàTmeNT iN germàNY. the SàFe àND eàSY-TO-USe scIg iNFUSiON TeChNiqUe [14] SUppOrTS scIg hOme TreàT-meNT àS àN àLTerNàTive TO IVIg TheràpY [15, 16]. a pOSSiBLe iNCreàSe iN qUàLiTY OFLiFe iS àNOTher im-pOrTàNT àSpeCT OFhOme-BàSeD scIg TreàTmeNT. oNLY LimiTeD DàTà ON SeLF-repOrTeD OUTCOmeS iN heàLTh-reLàT-eD qUàLiTY OFLiFe (HRQOl) iN àDULTS àND ChiLDreN WiTh primàrY àNTiBODY DeFiCieNCieS reCeiviNG repLàCemeNT TheràpY àre àvàiLàBLe [17, 18, 19, 20, 21]. HOWever,