He Mieloma Nui (MM)-01

ʻO ka hoʻihoʻi maikaʻi ʻana o kahi maʻi Myeloma i loko o ka hale me ka lāʻau lapaʻau CAR-T ʻoiai ʻaʻole i loaʻa ka BCMA Expression.

ʻO kahi maʻi wahine i loaʻa i ka pae IIIA IgD-λ type multiple myeloma i ka makahiki 2018 i loaʻa i ka lāʻau lapaʻau mua me ka bortezomib. Ma hope o 3 pōʻaiapuni, loaʻa iā ia ke kala piha (CR). I ʻOkakopa 2018, ua loaʻa ʻo ia i ka autologous hematopoietic stem cell transplantation ma ke ʻano he consolidation therapy, a ukali ʻia e ka mālama mālama me ka lenalidomide. I ʻApelila 2020, ua hoʻi hou ka maʻi, a ua loaʻa ʻo ia i 7 mau manawa o ka lāʻau lapaʻau lua, me ka maikaʻi ʻole. Mai Kekemapa 2020 a hiki i ʻApelila 2021, ua loaʻa ʻo ia i ka chemotherapy me ka daratumumab, akā ua hōʻike mau ʻia ka biopsy iwi iwi i ka 21.763% malignant monoclonal plasma cell, me ka serum free light chain λ ma 1470 mg/L a me ka urine free light chain λ ma 5330 mg/L. I kēia manawa, ua hoʻopau ʻo ia i nā lāʻau lapaʻau maʻamau a me nā mea hou i loaʻa ma ka home, me ka hoʻokomo ʻana i ka autologous stem cell transplantation, me ke komo ʻana i kahi hoʻokolohua lapaʻau CAR-T ʻo ia ka koho maikaʻi loa i koe.

Ua kuhikuhi ʻia e nā kauka kūloko, ua hōʻike ʻo ia i ka Halemai Ludaopei ma Mei 10, 2021, me ka manaʻo e kākau inoa i kā lākou hoʻokolohua lapaʻau no BCMA CAR-T therapy i ka myeloma lehulehu (MM). Ma ke komo ʻana, ua nāwaliwali ʻo ia me ka ʻeha nui a me ke kuni mau. Ua hōʻoia ʻia nā hoʻokolohua holoʻokoʻa "mieloma lehulehu, λ ʻano kaulahao māmā, ISS pae III, R-ISS pae III, mSMART pūʻulu pilikia nui."

Ua hōʻike ʻo PET-CT scan i ka hoʻonui ʻana i ka hana metabolic i loko o ka ʻiʻo palupalu palupalu i loko o nā lua iwi iwi o nā femurs bilateral a me tibias, e hōʻike ana i ke komo ʻana o ka maʻi maʻi. Ua hōʻike ʻia ka biopsy o ka iwi iwi i ka 60.13% malignant monoclonal plasma cell me ka ʻole o ka hōʻike ʻana o BCMA.

Ua haʻi aku ka Halemai Ludaopei i ka mea maʻi a me kona ʻohana e pili ana i ka maikaʻi o nā lāʻau lapaʻau i kēia manawa no ka BCMA-negative multiple myeloma, ʻoiai ʻo ia ka mea hiki ke kūpono e like me kekahi mau palapala, ʻaʻohe ʻikepili paʻa. Ma hope o ka noʻonoʻo pono ʻana, ua koho ka mea maʻi a me kona ʻohana e hoʻomau i ka papahana lapaʻau.

Ma hope o ka preconditioning me FC regimen, ua hoʻokomo ʻia nā cell BCMA CAR-T ma Iune 1, 2021, ma ka Halemai Ludaopei. Ua hoʻomohala ka mea maʻi i ke kuni ma hope o ka infusion, i hoʻomalu mālie ʻia me ka hoʻomaʻamaʻa anti-infective a me ka mālama kākoʻo symptomatic. He ʻumikūmāhā mau lā ma hope o ka hoʻokomo ʻana, ʻaʻole i hōʻike ʻia ka biopsy o ka iwi iwi i ke koena o nā pūnaewele plasma monoclonal malignant. He kanakolukūmākahi mau lā ma hope o ka hoʻokomo ʻana, ʻaʻole maikaʻi ka biopsy o ka iwi iwi. ʻAʻole maikaʻi ka serum immunofixation, serum free light chain λ i loko o ka laulā maʻamau, a ʻaʻole maikaʻi ka serum M protein, e hōʻike ana i ke kala piha ʻana o ka maʻi.

I kēia manawa, ʻoi aku ma mua o 8 mau mahina ma hope o ka loaʻa ʻana o ka BCMA CAR-T cell infusion, noho ka mea maʻi i ke kala piha me ka hoʻōla maikaʻi a me ka hauʻoli kiʻekiʻe i ka hopena o ka mālama ʻana.