Chithandizo cha cannabinoid pa ululu wonse wa EB ndi kuyabwa (2024)

Chiyesochi chinayesa mafuta otchedwa Transvamix, omwe ali ndi cannabis yachipatala, kuti awone ngati amachepetsa ululu mu EB. Akuluakulu asanu ndi atatu (amuna ndi akazi opitirira zaka 16) omwe ali ndi EBS, JEB kapena DDEB anamaliza phunziroli podzipangira okha mafuta pansi pa lilime lawo. Sanafunikire kusiya mankhwala ena opweteka omwe amamwa ndipo amatha kuwonjezera kugwiritsa ntchito mafuta kwa milungu iwiri mpaka atathandizira, kapena adawona zotsatirapo zake. Ena adapatsidwa Transvamix poyambirira, kenako adasinthidwa ndi mafuta omwe amawoneka, amanunkhiza komanso amalawa zofanana koma analibe chamba chachipatala (placebo). Ena anapatsidwa mafuta a placebo poyamba kenaka kusinthidwa kupita ku Transvamix. Ngakhale anthu omwe akutenga nawo mbali mu phunziroli kapena ochita kafukufuku sankadziwa, panthawiyo, ngati akulandira Transvamix kapena placebo ndipo dongosolo linasankhidwa mwachisawawa kwa munthu aliyense (kuyesa kosasintha, kawiri kawiri).

Mlanduwu udatenga milungu isanu ndi iwiri kwa munthu aliyense, panthawi yomwe adamaliza mafunso okhudza zizindikiro zawo za EB ndipo anali ndi MRI yaubongo wawo. Kafukufukuyu adamaliza kumapeto kwa 2024 ndikuwonetsa kuti Transvamix idapereka maubwino ena. Zinachepetsa ululu wa neuropathic, ndipo theka la anthu omwe amamwa mankhwala ena opweteka anasankha kuchepetsa kapena kuimitsa palimodzi pogwiritsa ntchito Transvamix. Zotsatira zonse zomwe zafotokozedwazo zidathetsedwa paokha popanda chithandizo chamankhwala kapena kulowererapo. Zotsatira zidzawunikidwa kwathunthu ndikufalitsidwa.

Mu Novembala 2023, ofufuza adanenanso kuti amalembetsabe maphunziro azachipatala. Akufunika anthu osachepera asanu ndi atatu kuti atenge nawo mbali kuti zotsatira zake zikhale zomveka. Mwa anthu asanu ndi awiri omwe akutenga nawo mbali pakali pano, oposa theka ndi omwe amaliza kafukufukuyu. Akuyembekeza kulembetsa munthu wachisanu ndi chitatu koyambirira kwa 2024 ndipo adzasanthula zotsatira anthu onse asanu ndi atatu akamaliza kafukufukuyu.

Mu Epulo 2023, ofufuza adanenanso kuti kulembera odwala kuti achite kafukufukuyu kudayamba mu Marichi 2022 koma ndi anthu ochepa omwe anali oyenera kuposa momwe amayembekezera chifukwa cha ma scan a MRI omwe adakonzedwa. Anasintha ndondomeko yawo kuti anthu omwe sangakhale ndi ma scan a MRI athe kutenga nawo mbali pa kafukufukuyu. Odwala asanu ndi awiri akuyembekezeka kumaliza kafukufukuyu kumapeto kwa Ogasiti 2023.

Ofufuza adasindikiza protocol yawo yoyeserera mu Disembala 2022 ndipo adalandira mayankho angapo. Amalimbikitsa kufalitsa zolembedwa pamanja zofanana ndi magulu ena ofufuza m'munda wa EB.

Zosintha zidagawidwa ndi bungwe mu Marichi 2022:

UMCG imayamba kufufuza za zotsatira za mafuta a cannabis mwa odwala EB

Prof Dr André Wolff ndi mutu wa UMCG Pain Center, mpando wa UMCG Pelvic Pain Center ndipo ali ndi chidwi chapadera pa (zachipatala) zatsopano za chisamaliro cha odwala opweteka kwambiri. Ntchito yake imakhudzana ndi kuzindikiridwa kolondola kwa ululu ndi njira zowononga odwala opweteka kwambiri komanso kuzindikira ululu wa neuropathic. Amagwiranso ntchito pakuchita sayansi komanso chisamaliro chaumoyo.

Dr Marieke Bolling. Anateteza bwino chiphunzitso chake cha PhD mu 2010 chotchedwa: Epidermolysis Bullosa Simplex. Zomwe adakumana nazo pakufufuza ndi kuyang'anira zidzakhala zofunikira pakukhazikitsa, kukhazikitsa ndi kutsiriza kwa polojekitiyi.

Dr José Duipmans, wakhala akudzipereka kuti apititse patsogolo chisamaliro kudzera mukulankhulana kwachangu tsiku ndi tsiku ndi odwala komanso nthambi ya DEBRA NL. Nthawi yochuluka ya nthawi yake yakhala ikugogomezera kumvetsetsa zosowa za odwala, ziyembekezo za banja ndi kumvetsetsa mavuto akuluakulu omwe ana omwe ali ndi EB amawaona. José atenga nawo gawo pazokhudza odwala paphunziroli ndikupereka mfundo yofunika kwambiri yolumikizirana mu phunziroli.

Nicholas Schräder, BSc, wakhala akugwira ntchito ndi EB kuyambira 2010 ndipo wakhala akugwira ntchito mwakhama mu mapulogalamu a maphunziro okhudzana ndi EB ndi maphunziro kuphatikizapo zochitika ku Chinese University of Hong Kong Prince of Wales Hospital, EB Research Center University of Freiburg Medical Center, Great Ormond Street Chipatala cha Ana ndi University Medical Center ya Groningen. Kuyambira 2012, Nicholas wakhala akuchita nawo kafukufuku wa sayansi kwa EB motsogozedwa ndi Prof. Marcel Jonkman, poganizira za chisamaliro chapadera komanso moyo wabwino. Kukumana ndi nthano zosawerengeka zakugwiritsa ntchito mankhwala osokoneza bongo pamavuto osiyanasiyana okhudzana ndi EB, ndikugwira ntchito mkati mwa dongosolo lazachipatala la Dutch lomwe lili ndi EB-center yokhazikitsidwa yamitundu yosiyanasiyana, adatha kuwunikanso zomwe zingachitike ndi chamba chachipatala, kapena cannabinoids, kwa anthu. akudwala EB.

Prof Dr Marcel Jonkman anamwalira mu Januwale, 2019. Iye anali wotchuka chifukwa cha njira yake yopangira kafukufuku wa sayansi ndi maphunziro a EB. Ntchito yake ndi utsogoleri wake zinapangitsa kuti amvetsetse mozama za miyeso yambiri yomwe imagwira ntchito mu matenda a EB, mkati ndi kunja kwa chipatala.

"Nkhani, nkhani ndi mafunso ochokera kwa odwala zakhala zofunikira kwambiri pakumvetsetsa kwathu kwanthawi yayitali kwamankhwala a cannabinoid omwe amagwiritsidwa ntchito pazizindikiro za EB ... Yakwana nthawi yoti tiwunike mwasayansi chithandizo chamankhwala chodziwika bwino ndikugwiritsa ntchito njira zachipatala zomwe zingasinthe kwambiri. moyo wa odwala athu omwe akuvutika ndi moyo wawo wonse. "

Mayina a Grant:

Kutsimikizira mphamvu ya sublingual phyto-cannabinoid yochokera mafuta ochizira ululu ndi pruritus (kuyabwa) mu epidermolysis bullosa.

Kafukufuku woyembekezeredwa wowongolera pa cannabinoids kuchiza kupweteka kosatha mu epidermolysis bullosa.

Transvamix (10% THC / 5% CBD) yochiza kupweteka kosalekeza mu epidermolysis bullosa: kafukufuku wosasinthika, wotsogozedwa ndi placebo komanso wakhungu wachiwiri.

Anthu omwe ali ndi epidermolysis bullosa (EB) amakumana ndi zizindikiro zosalekeza, zofooketsa, monga kupweteka ndi kuyabwa. Malipoti osawerengeka ochokera kwa odwala EB akuwonetsa kuti mankhwala opangidwa ndi cannabinoid (CBMs) ndi othandiza pakuwongolera zizindikiro.

Aliyense ali ndi dongosolo la endocannabinoid la mamolekyu ndi zolandilira zomwe zimagwira ntchito zosiyanasiyana za thupi, kuphatikizapo kuwonetsa ululu ndi kuyabwa - kupangitsa ubongo kuzindikira zizindikirozo.

Umboni wa matenda ambiri opweteka komanso oyabwa, komanso zomwe odwala a EB adakumana nazo, zikuwonetsa kuti ma CBM, opangidwa kunja kwa thupi (monga kuchokera ku chomera cha Cannabis) amafika pamlingo wocheperako pakuchepetsa zizindikiro ndi mankhwala wamba. Nthawi zambiri mankhwala oposa amodzi amagwiritsidwa ntchito pofuna cholinga chimodzi (monga kupweteka, opiates & anti-inflammatories amagwiritsidwa ntchito) zomwe zingayambitse zotsatira zosafunika kapena zosafunikira. Malipoti aposachedwa a odwala omwe ali ndi EB padziko lonse lapansi, ofotokoza zomwe adakumana nazo ndi ma CBM, onse olembedwa komanso odzipeza okha, akhala ochulukira, ndikuyitanitsa kafukufuku wasayansi kuti awone chitetezo ndi magwiridwe antchito a CBM. Ma CBM akupezeka m'maiko ochulukirachulukira, kuphatikiza Netherlands, komabe chidziwitso ndi malangizo a EB akusowa.
Choncho, pofufuza chithandizo chomwe chingakhalepo, polojekitiyi ikufuna kudziwa ngati ingathe kusintha moyo wa odwala omwe ali ndi EB.

Padziko lonse lapansi, ofufuza pano akuyang'ana mitundu yosiyanasiyana ya CBM ndi mitundu yoyang'anira matenda ambiri. Ku Netherlands kupita patsogolo kwaposachedwa kwapangitsa kuti mbewu za cannabis zizigwiritsidwa ntchito pochiza ndi odwala aku Dutch. Pankhani ya chisankho cha CBM pa kafukufukuyu, "phytocannabinoids" (zomera zochokera ku cannabinoids) zimachotsedwa muzomera ndikuphatikizidwa mumafuta omwe amaperekedwa ngati madontho pansi pa lilime (sublingual). Chinthu chomaliza ndi mankhwala - grade CBM mafuta, ndipo panopa amaperekedwa kwa odwala ku Netherlands omwe adalandira mankhwala kuchokera kwa dokotala wawo wolembetsa.

Kugwiritsa ntchito ma CBM opangidwa ndi zomera sikusokoneza thupi, ndipo sikunaphatikizidwe ndi zoopsa kapena zofooketsa. Popeza pakadali pano palibe chidziwitso chokwanira chokhudza zotsatira za nthawi yayitali za kugwiritsidwa ntchito kwa cannabinoid pakupanga dongosolo lamanjenje mwa ana, kafukufukuyu ndi wa odwala opitilira zaka 18 okha. Kuphatikizika kwapadera kudzaganizira za matenda amisala omwe analipo kale chifukwa izi zitha kukhudzidwa ndi kugwiritsa ntchito CBM.

Kafukufukuyu adzafufuza ngati mankhwala owonjezera ndi mafuta a sublingual awa angapangitse moyo wa anthu akuluakulu omwe ali ndi EB akuvutika ndi ululu ndi / kapena kuyabwa. Zotsatira za mafuta a CBM zimatha kukhala maola 4-8 motero ziyenera kutengedwa nthawi 4 tsiku lililonse kuti musunge magazi. Odwala adzanena zowawa, kuyabwa ndi kusintha kwa moyo wawo mwezi uliwonse pa miyezi 6. Ubwino wa moyo, ululu ndi kuyabwa zidzayesedwa pogwiritsa ntchito mafunso a odwala kapena miyeso ya zotsatira za odwala. Zambiri mwa izi zidzagwiritsidwa ntchito kuti zizindikirenso kuti ndi miyeso iti yomwe imakhudzidwa kwambiri ndi kusintha kwa odwala omwe ali ndi EB.
Kuyimitsa mankhwala omwe alipo panopa sikofunikira ngati wophunzira, panthawi ya kafukufuku, gulu lofufuza lidzayang'anira kusintha kwa mankhwala-kugwiritsa ntchito kwa wodwala aliyense (monga kuchepetsa kugwiritsa ntchito opiate), ndipo pamapeto pake fufuzani ngati izi zikugwirizana. kugwiritsa ntchito mafuta a CBM.

Kafukufukuyu amaonedwa kuti ndi oyembekezera, chizindikiro chotseguka, umboni wa phunziro la lingaliro - kuyesa mankhwala atsopano mwa odwala ochepa, kumene aliyense amamwa mankhwala atsopano kuti awone zomwe zingathe kudziwika. Choncho, pofufuza chithandizo chomwe chingakhalepo, polojekitiyi ikufuna kudziwa ngati ingathe kusintha moyo wa odwala omwe ali ndi EB.

Zokumana nazo zabwino zomwe azachipatala komanso odwala omwe ali ndi EB omwe ali ndi cannabinoids amagwiritsidwa ntchito ngati achire, zathandizira kwambiri kumvetsetsa kwathu. Phunziroli lidzayamba ndondomeko yowunikira mwasayansi zochitikazi ndikugwira ntchito ku ndondomeko ya chithandizo ndi malangizo okhudzana ndi umboni omwe angasinthe kwambiri moyo wa odwala EB. Monga momwe kafukufukuyu akukhudzira kugwiritsa ntchito mafuta a CBM omwe amayendetsedwa ndi chilankhulo, zotsatira zake ndi ziganizo zidzakhala zogwirizana kwambiri ndi mawonekedwe a CBM ndi kayendetsedwe ka kayendetsedwe ka kayendetsedwe kake, monga momwe mapangidwe ena ndi machitidwe oyendetsera ntchito amapangidwira ndi thupi la munthu pogwiritsa ntchito njira zosiyanasiyana.

Kafukufuku woyembekezeredwa wowongolera pa cannabinoids kuchiza kupweteka kosatha mu epidermolysis bullosa.

Transvamix (10% THC / 5% CBD) yochiza kupweteka kosalekeza mu epidermolysis bullosa: kafukufuku wosasinthika, wotsogozedwa ndi placebo komanso wakhungu wachiwiri.

Ntchito yokonzekera ndi yoyendetsera mayesero achipatala yatha. Kuphatikiza pa kuvomereza zamakhalidwe, njira zoyendetsera mankhwala ophunzirira, kujambula kwa radiographic ndi mafomu a lipoti lamilandu pa intaneti (database) akhazikitsidwa ndikuvomerezedwa. Pofika pa 15-03-2022 kulembera anthu kuti atenge nawo mbali kwayamba. Nthawi yoyembekezeka yophatikizidwa ndi miyezi 6. Kutsekedwa komwe kukuyembekezeka kutsatiridwa ndi kusanthula kwa data ndi Q4 2022.

Gulu lofufuza lidakumana ndi kuchedwa kwakukulu chifukwa cha kuwunika koyenera. Izi zinapangitsa kuti kukonzanso njira zoyesera zachipatala kupititsa patsogolo ubwino ndi mphamvu za phunzirolo - motsatira ndondomeko ya deta (fMRI-use, placebo control, cross-over design). Kuphatikiza apo, zovuta zonse zozungulira mliri wa COVID, ndi malamulo otsatila, zidapereka kuchedwa kwina. Monga taonera pamwambapa, kusintha kwa njira kumachepetsa kutenga nawo mbali, ndikulola kusonkhanitsa ndi kusanthula zotsatira. Popeza kuvomerezedwa kuti ayambe ntchito zofufuza, chidwi cha atolankhani chakula, DEBRA-UK amadziwika kuti ndi omwe amagawa zopereka. (Kuchokera ku lipoti la 2022).

Introduction

Phunziro la C4EB linatha kumapeto kwa 2024. Ntchitoyi inayambitsidwa ndi gulu lathu lofufuza ku University Medical Center ya Groningen, ndikuyang'ana pa kafukufuku yemwe amawongolera moyo wa tsiku ndi tsiku. Gululi linaphatikizapo gulu la akatswiri osiyanasiyana: José Duipmans (Namwino Wothandizira), Marieke Bolling (Dermatologist), André Wolff (Katswiri Wopweteka), Roy Stewart (Epidemiologist), Karin Vermeulen (Methodologist), Peter Söros (Neurologist / Neurophysiologist), ndi Nicholas Schräder (Dokotala Wofufuza).

Kafukufuku wa C4EB anali ndi cholinga chofufuza ngati mankhwala opangidwa ndi cannabinoid, Transvamix (chamba chamankhwala mu mawonekedwe a mafuta opaka pansi pa lilime), angathandize kuchepetsa ululu kwa odwala Epidermolysis Bullosa (EB). Mankhwala ophunzirira adakonzedwa ndi malo opangira kafukufuku ndipo anali ndi ma cannabinoids awiri: THC ndi CBD. M'zaka zaposachedwa, kafukufuku wawonetsa phindu lomwe lingakhalepo cannabinoids pothana ndi ululu ndi kuyabwa m'malo osiyanasiyana, kuphatikiza EB. Ngakhale kuti odwala nthawi zambiri amafotokoza zotsatira zabwino, ndikofunikira kuti aphunzire mankhwalawa mosamalitsa kuti amvetsetse momwe amagwirira ntchito komanso momwe amagwirira ntchito.

Popeza kuti phunziroli linali lokhudzana ndi chithandizo chamankhwala, njira zokhwima zinalipo za omwe angatenge nawo mbali komanso kuyeza komwe angapite. Ophunzirawo anayenera kukhala akuluakulu a zaka za 16 kapena kuposerapo, ndipo amafunikira kumva ululu monga chizindikiro cha EB panthawi yophatikizidwa. Kafukufukuyu adatenga masabata a 7 kwa wophunzira aliyense, pomwe adalandira onse a Transvamix ndi mafuta a placebo (omwe amawoneka, amanunkhiza, ndi kulawa mofanana ndi mankhwala ophunzirira). Kuti atsimikizire zotsatira zodalirika, otenga nawo mbali kapena ochita kafukufuku sanadziwe ndondomeko yomwe Transvamix kapena placebo inaperekedwa.

Omwe adatenga nawo gawo pang'onopang'ono adawonjezera mlingo wawo pakadutsa milungu iwiri mpaka atapeza mpumulo kuzizindikiro zawo kapena kuzindikira zotsatira zoyipa. Kuphatikiza pa kumaliza kafukufuku wokhudza ululu ndi momwe adathanirana nazo, ophunzirawo adachitanso ma scan a MRI. Kujambula uku kunathandiza ochita kafukufuku kuona momwe ubongo umayendera zizindikiro zowawa, mofanana ndi kupanga "kujambula mavidiyo" a ntchito za ubongo. Pa nthawi yonse yophunzira, otenga nawo mbali adaloledwa kupitiriza mankhwala awo opweteka omwe amawagwiritsa ntchito nthawi zonse ndikusintha mlingo wawo ngati pakufunika.

Results

Odwala 9 omwe ali ndi EB adatenga nawo gawo mu kafukufukuyu. Tsoka ilo, wophunzira m'modzi adasiya msanga, ngakhale izi zinali zosagwirizana ndi Transvamix kapena placebo. Otsalawo adayimira mitundu yosiyanasiyana ya EB, kuphatikiza EB Simplex, Junctional EB, ndi Dominant Dystrophic EB.

Cholinga chachikulu cha phunziroli chinali kufufuza kowawa komwe kunagawa ululu mu mitundu inayi. Kwa atatu mwa mitundu iyi-kupweteka kosalekeza, kupweteka kwapakati, ndi kupweteka kwapamtima (kupweteka kwamaganizo) -panalibe kusiyana kwakukulu pakati pa mpumulo pakati pa placebo ndi Transvamix. Komabe, chifukwa cha ululu wa neuropathic (ululu wokhudzana ndi kusokonezeka kwa mitsempha), otenga nawo mbali adafotokoza mpumulo wochulukirapo akamagwiritsa ntchito Transvamix poyerekeza ndi placebo.

Kuonjezera apo, pakati pa anthu omwe anali kumwa mankhwala opweteka panthawi yophunzira, theka linatha kuchepetsa mlingo wawo kapena kusiya mankhwala awo palimodzi pogwiritsa ntchito Transvamix. Chofunika kwambiri, onse omwe adatenga nawo mbali komanso ochita kafukufuku adanenanso zotsatira zilizonse (zoyipa) zomwe zidachitika panthawi yophunzira, kaya zokhudzana ndi placebo kapena Transvamix. Zotsatira zonse zomwe zafotokozedwa zinkawoneka ngati zochepa kapena zochepa ndipo zinathetsedwa paokha, makamaka mkati mwa maola ochepa, popanda kufunikira chithandizo chamankhwala kapena kulowererapo.

Zomwe Zikuyenera Kukonzedwa

Deta ya MRI yomwe yasonkhanitsidwa panthawi yophunzira imafuna mawerengedwe ovuta komanso kusanthula mwatsatanetsatane, zomwe zikuchitikabe. M'masabata angapo otsatirawa, gulu lathu lidzamaliza zotsatira ndikukonzekera chidule chatsatanetsatane kuti chifalikidwe mu magazini ya sayansi, kulola ofufuza a EB ndi omwe si a EB padziko lonse lapansi kuti aphunzire kuchokera ku zomwe tapeza.

Mawuwo

Kafukufukuyu ndi woyamba kuyesa kugwiritsa ntchito mankhwala opangidwa ndi cannabinoids kuti athetse ululu kwa odwala omwe ali ndi EB. Ngakhale kuti zotsatirazo sizingaganizidwe ngati zoyambira, zimapereka chidziwitso chofunikira. Transvamix, ikagwiritsidwa ntchito m'malo olamulidwa ndi otetezeka, inasonyeza phindu lomwe lingakhalepo kwa odwala ena omwe ali ndi EB, makamaka poyang'anira ululu wa neuropathic. Kuchepetsa kugwiritsa ntchito mankhwala ena opweteka komanso kufatsa kwa zotsatirapo zomwe zanenedwa zimathandiziranso kuthekera kwamankhwala opangidwa ndi cannabinoid pa ululu wokhudzana ndi EB.

Tsogolo Labwino

Kupyolera mu phunziroli, tikuyembekeza kulimbikitsa ochita kafukufuku ena ndi asing'anga kuti afufuze zofananira, pochita zachipatala ndi kafukufuku. Wodwala aliyense yemwe ali ndi EB ndi wapadera, ndipo pali njira zambiri zodalirika zowonera chithandizo chamankhwala a cannabinoid. Izi zikuphatikizapo kusiyana kwa cannabinoids (CBD vs. THC), mitundu yoyendetsera (mafuta, kirimu, nthunzi), ndi kuthetsa mavuto ena omwe odwala amakumana nawo, monga kuyabwa, mabala aakulu, ndi zipsera. Kuonjezera apo, njira ya phunziroli-kugwiritsa ntchito gulu laling'ono la otenga nawo mbali ndikuphatikiza placebo-imapereka ndondomeko yodalirika ya kafukufuku wamtsogolo. Gulu lathu pakali pano likugwira ntchito ya COSEB (Core Outcome Set for EB), yomwe cholinga chake ndi kulinganiza zida ndi miyeso yomwe imagwiritsidwa ntchito mu kafukufuku wa EB. Ndife okondwa kuyankha mafunso aliwonse omwe mungakhale nawo okhudza kafukufukuyu. Chonde pirirani nafe pamene tikukonzekera kufalitsa deta yonse mwamsanga. Chonde onani maulalo pansipa, nkhani zonena za ntchito yathu.

Maulalo:

UMCG imayamba kafukufuku wasayansi mu mafuta a cannabis chifukwa cha ululu wosaneneka mu Epidermolysis Bullosa November 2021

Kuchita bwino kwamafuta a cannabis pakuwongolera ululu kwa odwala a EB

UMCG imayamba kufufuza za zotsatira za mafuta a cannabis mwa odwala EB March 2022

UMCG imayamba kafukufuku wamankhwala a cannabis a Epidermolysis Bullosa March 2022

UMCG imayamba kufufuza za zotsatira za mafuta a cannabis March 2022

(Kuchokera ku lipoti lomaliza la 2024.)