He aha te Lumasiran: Ngā Whakamahinga, te Inenga, ngā Pānga taha, me ētahi atu

He rongoā motuhake te Lumasiran i hangaia hei āwhina i te hunga e mate ana i tētahi āhua ira onge e kīia ana ko te hyperoxaluria tuatahi momo 1 (PH1). E mahi ana tēnei rongoā werohia mā te whakaiti i te nui o te oxalate e whakaputaina ana e tō tinana, ka āwhina pea i te aukati i ngā kōhatu whatukuhu mamae me te tiaki i ō whatukuhu mai i te kino.

Mēnā kua tātai koe, tētahi rānei e āwangawanga ana koe i te PH1, tērā pea kei te pōuri koe i ngā pātai me ngā āwangawanga. E pā ana tēnei āhua ki te āhua o te tukatuka a tō tinana i ētahi mea, e arahi ana ki te kohinga o te oxalate ka puta he raruraru whatukuhu nui. Ko te rongo pai ko te tohu a te lumasiran i tētahi whakatutukitanga nui i te rongoā i tēnei āhua uaua, e tuku ana i te tūmanako me te kounga o te oranga pai ake mō te maha o ngā tūroro.

He aha te Lumasiran?

He rongoā whakahau te Lumasiran e noho ana ki tētahi kāwai rongoā e kīia ana ko te RNA interference (RNAi) therapeutics. E mahi ana mā te

Kua whakaaetia a Lumasiran hei rongoā i te hyperoxaluria tuatahi momo 1 (PH1) i roto i ngā pakeke me ngā tamariki. He āhua ira tēnei momo mate onge e nui rawa ana te hanga oxalate e tō tinana, he matū ka puta he kōhatu whatukuhu mamae, ka whara i ō whatukuhu i te roanga o te wā.

I roto i ngā tāngata e pā ana ki te PH1, nā te huringa ira ka nui rawa te hanga oxalate e te ate, nā te mea kāore he enzyme nui e kīia ana ko te alanine-glyoxylate aminotransferase (AGT). Ki te kore tēnei enzyme e mahi tika ana, ka huri ngā matū e tika ana kia tukatukahia kia pai ki te oxalate. Ka haere tēnei oxalate nui ki roto i ō toto ki ō whatukuhu, ka taea te whakakoi me te hanga kōhatu.

Ka taea e tēnei āhua te arahi ki ngā raruraru nui ki te kore e rongoatia. Kei roto i ēnei ko ngā kōhatu whatukuhu e hoki mai ana, te whara o te whatukuhu, te korenga o te whatukuhu, me ngā take nui, he āhua e kīia ana ko te systemic oxalosis kei reira ngā tioata oxalate e tū ana i roto i ētahi atu okana pēnei i te ngākau, ngā wheua, me ngā kanohi. Ka āwhina a Lumasiran ki te ārai i ēnei raruraru mā te whakatika i te take o te nui o te hanga oxalate.

Me pēhea te mahi a Lumasiran?

Ka mahi a Lumasiran mā tētahi tukanga uaua e kīia ana ko te RNA interference, e kī ana ki ō pūtau ate kia mutu te hanga oxalate nui. Ka whai te rongoā i te ira LDHA, e kawenga ana mō te hanga i tētahi enzyme e whai wāhi ana ki te hanga oxalate.

I te wā e whiwhi ana koe i te werohanga lumasiran, ka haere te rongoā ki tō ate kei reira e here ana ki ngā ngota RNA karere motuhake. Ko ēnei ngota te tikanga e mau ana i ngā tohutohu mō te hanga pūmua e arahi ana ki te hanga oxalate. Mā te raru i ēnei karere, ka whakaiti a lumasiran i te nui o te oxalate e hangaia ana e tō tinana ki te 65% neke atu rānei.

He rongoā kaha tēnei e pā ana ki tōna whai huatanga, engari he mea whāinga nui anō hoki. Kāore i rite ki ētahi rongoā e pā ana ki ngā pūnaha maha o te tinana, ka arotahi te lumasiran ki ngā pūtau ate e kawenga ana mō te nui rawa o te hanga oxalate. Ka roa pea ngā pānga o ia werohanga mō ētahi marama, koinei te take kāore koe e hiahia ki te rongoā i ia rā, pērā i ētahi atu rongoā.

Me pēhea taku tango i te Lumasiran?

Ka tukuna te Lumasiran hei werohanga i raro i te kiri, arā, ka werohia ki roto i te kiko momona i raro tonu i tō kiri. Mā tō kaiwhakarato hauora koe, tō whānau rānei e ako me pēhea te tuku i ēnei werohanga i te kāinga, ahakoa e pai ana ētahi tāngata kia mahia ki tētahi whare rongoā.

Ko te tikanga ka tukuna te werohanga ki ngā wāhi e nui ana te momona i raro i te kiri, pērā i tō huha, tō ringa o runga, tō puku rānei. Ka huri koe i ngā wāhi werohanga hei ārai i te riri, i te whē rānei i tētahi wāhi. Ka tae mai te rongoā i roto i ngā syringe kua whakakiia, i ngā ipu rānei me waiho ki te pouaka whakamātao kia whakamahia ra anō.

Kāore i rite ki ngā rongoā waha, kāore koe e hiahia ki te māharahara mō te tango i te lumasiran me te kai, i runga rānei i te puku kau, nā te mea ka werohia. Heoi anō, me tuku te rongoā kia tae ki te pāmahana rūma mō te 30 meneti i mua i te werohanga kia pai ake ai. Ka hoatu e tō tākuta ngā tohutohu taipitopito mō te tikanga werohanga tika, te rokiroki, me te whakakore i ngā ngira me ngā syringe kua whakamahia.

Kia pehea te roa o taku tango i te Lumasiran?

Ko te tikanga he rongoā wā roa te Lumasiran e hiahia ana koe ki te haere tonu i ngā wā katoa. I te mea he āhuatanga ira te PH1, ka haere tonu tō tinana ki te whakaputa nui i te oxalate me te kore he rongoā e haere tonu ana hei pupuri i te tūnga.

Ko te mahere maimoatanga tuatahi ko te nuinga ka uru ki nga werohanga auau ake hei whakaiti tere i o taumata oxalate, ka whai muri i te horopeta tiaki ia toru marama. Ka aroturukihia e to taakuta o taumata oxalate mimi i nga wa katoa kia mohio kei te pai te mahi o te rongoa, a ka whakatika pea i to mahere horopeta i runga i to urupare takitahi.

Ko te nuinga o nga tangata ka timata ki te kite i te whakahekenga o o ratou taumata oxalate mimi i roto i te marama tuatahi o te maimoatanga, me nga paanga nui ka tutuki i muri i etahi marama o te horopeta rite tonu. Ka mahi tahi to roopu hauora ki a koe ki te whakarite i tetahi mahere maimoatanga mo te wa roa e pai ana ki to oranga i te wa e whakarato ana i te whakahaere mate pai.

He aha nga paanga taha o Lumasiran?

Pērā i ngā rongoā katoa, ka taea e te lumasiran te pānga taha, ahakoa he maha ngā tāngata e pai ana ki te tango. Ko nga paanga taha tino noa he ngawari noa iho, he hono ki te werohanga ake, kaua ki nga paanga punaha puta noa i to tinana.

Ko nga paanga taha e tino korerohia ana ko nga tauhohenga i te waahi werohanga, penei i te whero, te pupuhi, te patito, te mamae ngawari ranei i te waahi i whakauruhia ai te ngira. Ko enei tauhohenga he wa poto noa iho, ka ngaro noa iho i roto i etahi ra. Ka pa ki etahi tangata nga tohu ngawari penei i te rewharewha, tae atu ki te ngenge, te mahunga, te mamae uaua ranei, ina koa i muri i o ratou werohanga tuatahi.

Ko nga paanga taha kaore i te tino noa engari he nui ake te awangawanga ka uru pea ki nga tauhohenga mate pāwera, ahakoa he onge enei. Ko nga tohu o te tauhohenga mate pāwera nui ka uru pea ki te uaua ki te manawa, te pupuhi nui o to kanohi, korokoro ranei, te pupuhi whanui ranei. Mena ka pa ki a koe etahi o enei tohu, rapua tonu te rongoa.

Ka pātai pea ētahi tāngata i ngā huringa i roto i ā rātou whakamātautau mahi ate, koinei te take ka aroturukihia e tō tākuta ēnei taumata i ia wā. I ngā wā onge, ka pātai pea ngā tāngata i ngā raruraru tākihi, ahakoa he nui ake te hononga o tēnei ki te āhua PH1 kei raro iho i te rongoā ake. Ka tino aroturukihia e tō rōpū hauora tō mahi tākihi i te wā o te rongoā.

Ko wai kāore e tika kia tango Lumasiran?

I hangaia motuhake a Lumasiran mō ngā tāngata e pā ana ki te hyperoxaluria tuatahi momo 1, nō reira kāore i te tika mō ētahi atu momo mate kōhatu tākihi, ngā āhua hyperoxaluria rānei. Ka whakapūmau tō tākuta i tō tātaritanga PH1 mā te whakamātautau ira i mua i te whakahau i tēnei rongoā.

Kāore e tika kia whakamahia e ngā tāngata e tino pā ana ki te mate pāwera ki a lumasiran, ki ētahi rānei o ōna wāhanga. Mēnā he hītori tōu mō ngā tauhohenga pāwera nui ki ētahi atu rongoā werohia, me tino kōrero tēnei ki tō kaiwhakarato hauora i mua i te tīmatanga o te rongoā.

Mēnā he mate ate nui tōu, he mahi ate rānei kua tino pāngia, tērā pea ka hiahia tō tākuta ki te āta arotake mēnā he tika a lumasiran mōu, nā te mea ka mahi te rongoā i te ate. Waihoki, mēnā he mate tākihi nui tōu, kei te haere rānei koe i te dialysis, tērā pea ka hiahia tō mahere rongoā ki te whakaaro motuhake.

Me kōrero ngā wāhine hapū, ngā wāhine e whāngai ana i te ū ki ngā tāngata hauora mō ngā tūponotanga me ngā painga o lumasiran, nā te mea he iti noa ngā raraunga mō tōna haumaru i te wā o te hapū me te whāngai ū. Ka āwhina tō tākuta ki te whakatau i ngā painga pea ki ngā tūponotanga pea ki a koe me tō pēpi.

Ingoa Waitohu Lumasiran

Ka hokohoko a Lumasiran i raro i te ingoa waitohu Oxlumo i te United States me ētahi atu whenua. I whiriwhiria tēnei ingoa waitohu hei whakaatu i te kaupapa o te rongoā ki te whakaiti i ngā taumata oxalate i roto i ngā tāngata e pā ana ki te hyperoxaluria tuatahi.

He mea hanga a Oxlumo e Alnylam Pharmaceuticals, he kamupene e tohunga ana ki nga rongoa aukati RNA. I whakamanahia te rongoa e te FDA i te tau 2020, koinei te rongoa tuatahi i whakaaetia i hangaia hei whakatika i te take o te PH1, kaua ki te whakahaere i ona tohu.

I te wa e korero ana koe mo to maimoatanga ki nga kaiwhakarato hauora, ki nga kamupene inihua ranei, ka tohuhia pea e ratou te rongoa ma tona ingoa whanui (lumasiran) ranei tona ingoa waitohu (Oxlumo). E rua nga ingoa e tohu ana ki te rongoa kotahi, no reira kaua e raruraru ki te rongo koe i nga kupu rereke e whakamahia ana.

Nga Whakaaro ke atu ki a Lumasiran

I mua i te waatea o lumasiran, he iti noa nga whiringa maimoatanga mo te PH1, a ko te nuinga i aro ki te whakahaere i nga tohu, kaua ki te whakatika i te take. Ko nga huarahi tuku iho ko te nui o te wai, te aukati i te kai oxalate, me nga rongoa penei i te potassium citrate hei awhina i te aukati i te hanganga o te kohatu.

Mo nga tangata whai PH1 kino i whakawhanake i te ngoikore o te whatukuhu, ko te whakawhitinga ate te mea anake te whiringa maimoatanga. Ka taea e te whakawhitinga ate te rongoa i te PH1 na te mea ka whakakapi i nga pūtau ate he ki nga mea hauora e whakaputa ana i te enzyme ngaro. Heoi, he nui nga tupono o te whakawhitinga, me te hiahia ki nga rongoa aukati i te mate mo te wa katoa.

Ko etahi tangata whai PH1 ka whai hua ano mai i te whakawhitinga ate-whatukuhu mena he mate ate me te ngoikore o te whatukuhu. Heoi, he uaua enei tikanga, kaore e tika ana mo te katoa. Ko te waatea o lumasiran kua tino huri i te whenua maimoatanga, e tuku ana i tetahi whiringa iti ake te whakaeke ka taea te aukati i te hiahia mo te whakawhitinga i te nuinga o nga keehi.

Ko nga maimoatanga tautoko penei i te nui o te wai, nga whakarereketanga kai, me nga rongoa hei whakakoi i te mimi ka whakamahia tonu i te taha o lumasiran hei whakarato i te manaaki whanui mo nga tangata whai PH1.

He pai ake a Lumasiran i etahi atu maimoatanga PH1?

He ahunga whakamua nui a Lumasiran i runga i nga rongoa tuku iho mo te PH1 na te mea koinei te rongoa tuatahi e aro tika ana ki te take o te mate, kaua ki te whakahaere noa i ona tohu. Kāore i rite ki ngā rāhui kai me te nui ake o te wai, he iti nei ngā painga, ka taea e lumasiran te whakaiti i te hanga oxalate ma te 65% neke atu rānei.

Ki te whakataurite ki te whakawhitinga ate, he pai ake te whiringa rongoa a lumasiran, he iti ake te pokanga me te iti ake o ngā tūponotanga me ngā raruraru. Ahakoa ka taea e te whakawhitinga ate te rongoa i te PH1, e hiahia ana kia pokanga nui, kia mau tonu te rongoa immunosuppressive, me te tūponotanga o te whakakore me ētahi atu raruraru nui. Ka taea e Lumasiran te tangata ki te whakahaere i tō rātou āhuatanga i te wa e mau tonu ana tō rātou kounga o te oranga.

He painga ano hoki te rongoa i runga i ngā rongoa e pa ana ki ngā tohu pēnei i te potassium citrate, te nui ake rānei o te wai. Ahakoa ka taea e ēnei huarahi te ārai i te hanganga kōhatu ki ētahi tohu, kāore e aro ki te take o te nui o te hanga oxalate. Ka whakawhiwhia e Lumasiran te whakahaere mate whānui ake mā te whakaiti i te nui o te oxalate e hangaia ana e tō tinana i te tuatahi.

Heoi, ko te huarahi rongoa pai rawa atu e whakawhirinaki ana ki ō āhuatanga takitahi, tae atu ki te kaha o tō āhuatanga, tō hauora whānui, me ō hiahia whaiaro. Ka mahi tahi tō rōpū hauora ki a koe ki te whakatau i te mahere rongoa e tika ana mō tō āhuatanga motuhake.

Ngā Pātai Auau mō Lumasiran

He haumaru a Lumasiran mō ngā tamariki?

Āe, kua whakaaetia a lumasiran mō te whakamahi i ngā tamariki, ā, kua whakaaturia he haumaru, he whai hua i roto i ngā tūroro tamariki e mate ana i te PH1. Kua akona te rongoa i roto i ngā tamariki e 6 tau te pakeke, ā, ka whakatikahia te inenga i runga i te taumaha o te tinana kia pai ai te taumata rongoa.

He maha ngā wā ka pai te urupare a ngā tamariki ki te rongoā lumasiran, me te whakahekenga nui o ngā taumata oxalate i roto i te mimi, ka āwhina i te ārai i ngā kōhatu whatukuhu me te tiaki i ō rātou whatukuhu e tipu ana. Me whakarerekē pea te tikanga werohanga mō ngā tamariki nohinohi, ā, ka whiwhi ngā mātua, ngā kaitiaki rānei i te whakangungu whānui mō te pēhea te whakahaere i te rongoā i runga i te haumaru.

Me mātakitaki tōtika ngā tūroro tamariki i te wā katoa o te rongoā, tae atu ki ngā whakamātautau mimi i ngā wā katoa hei tirotiro i ngā taumata oxalate me ngā whakamātautau toto hei aroturuki i te mahi o te whatukuhu me te ate. Ka whakatika te rōpū hauora o tāu tamaiti i te mahere rongoā i a rātou e tipu ana, e whanake ana hoki, hei whakarite i te whai huatanga me te haumaru tonu.

Me pēhea taku mahi mēnā ka whakamahi au i te nui rawa o te Lumasiran?

Mēnā ka werohia e koe te nui rawa o te lumasiran i te mea i whakaritea, whakapā atu ki tō kaiwhakarato hauora tonu, waea atu rānei ki te mana whakahaere paitini. Ahakoa he onge ngā take o te horopeta nui ki te lumasiran, he mea nui kia pūrongo i ngā hē horopeta kia taea ai e tō rōpū hauora te aroturuki i a koe i te tika.

Kaua e ngana ki te utu mō te horopeta nui mā te karo i tō horopeta e whai ake nei, ki te kore koe e tohutohutia e tō kaiwhakarato hauora. Ka hiahia pea rātou ki te aroturuki i ō whakamātautau mahi ate, i ngā taumata oxalate rānei i muri i te horopeta nui hei whakarite i tō haumaru.

Hei ārai i ngā horopeta nui ohorere, me tirotiro tonu i tō horopeta i mua i te werohanga, me te pupuri i tō rongoā kia tapaina tika, kia penapena tika. Mēnā kāore koe i te mōhio mō tō mahere horopeta, he pātai rānei āu mō te nui o te werohanga, whakapā atu ki tō kaiwhakarato hauora i mua i te haere tonu i te werohanga.

Me pēhea taku mahi mēnā ka ngaro au i tētahi horopeta o Lumasiran?

Mēnā ka ngaro koe i tētahi werohanga lumasiran i whakaritea, whakapā atu ki tō kaiwhakarato hauora i te wā e taea ai hei whakatau i te huarahi pai rawa atu. I te nuinga, me tango e koe te horopeta i ngaro i te wā e mahara ana koe, ka hoki anō ki tō mahere horopeta auau.

Kaua e rua ngā horopeta hei whakakī i tētahi werohanga i ngaro, tērā pea ka piki ake te tūponotanga o ngā pānga taha. Ka taea e tō tākuta te whakatika i te wā o tō horopeta e whai ake nei kia hoki ai koe ki te ara tika me tō mahere maimoatanga.

E kore pea te ngaro i tētahi horopeta e pā ana ki ngā raruraru tonu, engari ngana ki te pupuri i tō mahere werohanga i te wā e taea ana mō te whakahaere pai o te mate. Whakaarohia te whakatakoto i ngā whakamaharatanga ki tō waea, ki tō maramataka rānei hei āwhina i a koe kia noho tika ki tō mahere maimoatanga.

Āhea ka taea e au te whakamutu i te tango i te Lumasiran?

Kaua rawa koe e whakamutu i te tango i te lumasiran me te kore e kōrero tuatahi ki tō kaiwhakarato hauora. I te mea he āhua ira te PH1, ka whakaputa tonu tō tinana i te oxalate mō te nui rawa mēnā ka whakamutua e koe te maimoatanga, ka taea e tērā te whakahoki mai i ngā kōhatu tākihi me te kino pea o te tākihi.

Ka taea e tō tākuta te whakaaro ki te whakatika i tō mahere horopeta mēnā kei te pai rawa koe i te maimoatanga, engari he uaua te whakamutu katoa. Mēnā kei te pā koe i ngā pānga taha e raruraru ana, he āwangawanga rānei mō te haere tonu i te maimoatanga, kōrero tuwhera ki tō rōpū hauora mō ō whiringa.

I ētahi wā, mēnā kei te whakamahere koe ki te whai whakawhitinga ate, ka taea e tō tākuta te kōrero mō te wā o te maimoatanga lumasiran huri noa i te tukanga. Heoi, me whakatau tonu ēnei whakataunga i te kōrero tahi me tō rōpū whakawhitinga me ngā tohunga PH1.

Ka taea e au te haereere me te Lumasiran?

Āe, ka taea e koe te haereere me te lumasiran, engari me whai mahere i te mea me pupuri te rongoā i roto i te pouaka whakamātao. I te haereere, whakakīhia tō rongoā ki roto i te pouaka mō te makariri me ngā pākete huka, me te mau i tētahi reta mai i tō tākuta e whakamārama ana i tō hiahia mō te rongoā.

Mēnā kei te rere koe, waiho tō lumasiran ki roto i tō pēke kawe i te wāhi i te pēke i tirohia kia kore ai e tio i roto i te wāhi utanga. Tērā pea me tirotiro e te haumarutanga o te rererangi tō rongoā, nā reira me whai wā anō mō te tātari me te kawe i ngā tuhinga mō tō rongoā.

Mō ngā haerenga roa, rangahaua ngā whare rongoā kei tō ūnga i te wā e hiahia ana koe ki te āwhina i tō werohanga, kei a koe rānei ētahi āwangawanga rongoā. Whakaarohia te mau i ētahi atu taonga hei ārai i ngā whakaroa, ā, me mōhio kei a koe te rongoā e nui ana mō tō haerenga katoa me ētahi rā anō.