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Unyango lweGene olutsha lubonelela ngeThemba elitsha kwiSickle Cell kunye nezigulana zeThalassemia

I-FUCASO: Unyango lwe-BCMA CAR-T yoMntu ngokuPheleleyo ngokuSebenza ngoKusebenza kunye noKhuseleko.

I-melanoma ye-Ocular (ekuqaleni), ilandelwa ngamathumba esibindi se-metastatic-02

Inqanaba eliphezulu le-IV lomhlaza we-colorectal-01

Umhlaza we-prostate womgangatho ophezulu uhamba kunye ne-bone metastases-05

I-Acute Lymphoblastic Leukemia(B-ALL)-02

Umonde: Wang XX

Isini:Mfazi

Ubudala: iminyaka emi-3 ubudala

Ubuzwe: IsiTshayina

UxilongoI-Acute Lymphoblastic Leukemia (B-ALL)

Qhagamshelana nathi

Iimpawu zeMpahla:

- Meyi 19, 2019: Ufunyanwe une-acute B-cell lymphoblastic leukemia (B-ALL)

-Ibonakaliswe ngobuninzi be-scalp kunye ne-lymphadenopathy

- Inkqubo yegazi: WBC 13.3 x 10^9/L, HGB 94 g/L, PLT 333 x 10^9/L, i-lymphocyte engaqhelekanga 4%

- I-morphology yomongo wethambo: i-80.2% ye-lymphoblasts engekavuthwa (iziqhushumbisi)

- I-Immunophenotyping: I-74.19% yeeseli ziyingozi ze-B-lineage precursor cells ezibonisa i-CD45dim, i-CD19, i-CD9, i-CD22, i-CD81, i-CD58, i-cCD79a, i-CD38, i-HLA-DR, i-cIgM ichaza ngokuyinxenye. Uxilongo: B-ALL (Inqanaba langaphambi kwe-B)

-Fusion gene: MLL-ENL positive, Philadelphia chromosome-like (Ph-like) screen negative

- Chromosome: 46, XX, t(11;19)(q23;p13), del(20)(q12) [3]/46, XX [7]

- I-VDLD yerejimeni ye-chemotherapy ekuqaleni ifumene ukuxolelwa kwe-immunological emva kwenyanga ye-1, i-MLL-ENL yobuninzi be-PCR 0.026%

- I-chemotherapy eqhubekayo njengeprotocol yabantwana, i-MLL-ENL yobuninzi be-PCR 0 emva komjikelezo we-4. Unyango olongezelelekileyo lwaqhubeka.

NgoMatshi ka-2020: Isifo se-Bone marrow immunological residual disease 0.35%, MLL-ENL quantitative PCR 0.53%, ebonisa umkhwa wokubuyela umva. Usapho lwala ukutshintshwa. I-chemotherapy eqhubekayo kwimijikelo emi-3.

-Julayi 2020: Umongo wethambo uphinde wabuyela ngokupheleleyo.

- Novemba 11, 2020: Intrathecal chemotherapy, CSF immunological residual disease 66%, kwafunyaniswa ukuba une-central nervous system leukemia. I-Intrathecal chemotherapy yaphinda kabini, i-CSF yajika yaba mbi.

-Disemba 31, 2020: Wangeniswa esibhedlele.

- Inkqubo yegazi: WBC 3.99 x 10^9/L, HGB 66 g/L, PLT 57 x 10^9/L

-Ubalo lwegazi elijikelezayo: 69%

- I-morphology yomongo wethambo: i-90% ye-lymphoblasts engekavuthwa (ukuqhushumba)

- I-Immunophenotyping: I-84.07% yeeseli zivakalisa i-CD38, i-CD19, i-CD81dim, i-cCD79a, i-HLA-DR, i-cIgM, i-CD22, i-CD123, i-CD24, i-CD15dim, ibonisa i-lymphoblasts enobungozi B engakafiki.

-Ijini ye-Fusion: I-MLL-ENL i-fusion gene positive, i-PCR yobungakanani be-44.419%

-Uguqulo lwemfuza: KMT2D uguqulo olulungileyo (imvelaphi yentsholongwane)

- Chromosome karyotype: 46, XX, del(1)(p36.1), del(1)(q31q42), del(11)(q13), t(11;19)(q23;p13.3), yongeza( 14)(q34), -17, +mar [7]/46, idem, t(3;16)(p21;p13.3) [1]/46, XX [13]

- I-PET-CT: Ukusabalalisa ukwanda kwe-metabolic kuwo wonke ama-skeletal and bone marrow cavity, ukukrokra okuphezulu kokuphindaphinda kwe-leukemia; i-splenomegaly kunye nokwanda kwemetabolism, mhlawumbi ibandakanya i-leukemia.

-Yenziwe i-lumbar puncture kunye ne-intrathecal chemotherapy kanye, akukho kuphazamiseka kufunyenwe kwiimvavanyo ezinxulumene ne-CSF.

Unyango:

Iiveki ezimbini ze-VLP chemotherapy, igazi le-peripheral liqhuma i-5% ngoJanuwari 18.

- NgoJanuwari 25: I-peripheral blood blasts 91%, iphathwa nge-CTX, i-Ara-C, i-6-MP i-chemotherapy.

- Februwari 3: Ukuqhuma kwegazi kwi-peripheral 22%.

- NgoFebruwari 4: Ukuqokelelwa kwe-50ml yegazi le-peripheral autologous kwinkcubeko ye-CD19-CART yeseli.

- I-MTX 1g, i-FC chemotherapy (i-Flu 15mg imihla ngemihla x iintsuku ze-3, i-CTX 0.12g imihla ngemihla x iintsuku ze-3).

- NgoFebruwari 13 (ngaphambi kokufakelwa): I-Bone marrow morphology ibonisa i-87.5% yokuqhuma, i-cytometry yokuhamba ibonisa i-79.4% yokuqhuma okubi.

- Uhlalutyo lwe-MLL-ENL ye-fusion ye-gene quantitative: 42.639%.

- NgoFebruwari 14: Ukufakwa kweeseli ze-CART kwi-dose ye-5 x 10 ^ 5 / kg.

- Imiphumo emibi enxulumene ne-CAR-T: IBanga le-1 CRS (umkhuhlane), akukho neurotoxicity.

- Usuku lwe-20 emva kokunyuswa: I-blood smear ibonisa ukwanda kwe-tumor, i-CART cell proportion 0.07%.

- Unyango lweeseli ze-CART olungasebenziyo.

- Matshi 8, 2021: Inkqubo yegazi: WBC 38.55 x 10^9/L, HGB 65g/L, PLT 71.60 x 10^9/L.

- Ukuqhuma kwegazi kwi-peripheral: 83%. Igazi le-autologous peripheral liqokelelwe i-60ml ye-CD19/CD22 inkcubeko yeseli ye-CART emibini.

- Ukunyangwa nge-cytarabine kunye ne-dexamethasone ukulawula umthwalo we-tumor.

- Matshi 18: I-FC chemotherapy (i-Flu 15mg imihla ngemihla x iintsuku ze-3, i-CTX 0.12g imihla ngemihla x iintsuku ze-3).

- Matshi 22 (pre-infusion): Inkqubo yegazi: WBC 0.42 x 10^9/L, HGB 93.70g/L, PLT 33.6 x 10^9/L. I-peripheral blood morphology: i-6% yokuqhuma.

- I-morphology yomongo wethambo: 91% ukuqhuma. Intsalela kumongo wethambo: 88.61% iiseli ezibonisa i-CD38, CD19, cCD79a, CD81, CD22, ebonisa i-lymphoblasts ye-B engalunganga.

- Uhlalutyo lwe-MLL-ENL ye-fusion ye-gene quantitative: 62.894%.

- Uhlalutyo lwe-Chromosome karyotype: 46, XX, del (1) (p36.1), del (11) (q13), t (11; 19) (q23;p13.3), yongeza (14) (q34), - 17, +mar [2]/46, XX, del(1)(p36.1), del(1)(q31q42), del(11)(q13), t(11;19)(q23;p13.3) ), yongeza(14)(q34).

- Matshi 23: Ukufakwa kweeseli ze-CART kwi-dose ye-3 x 10 ^ 5 / kg.

- Matshi 26 ukuya phambili: Umkhuhlane ongapheliyo kwaye emva koko waphuhliswa yi-systemic edema.

- Matshi 29: I-peripheral blood morphology: 92% ukuqhuma; ukwanda kwe-bilirubin kunye ne-transaminases.

Ngo-Epreli 2: Ukuqala kokuxhuzula, ukuphathwa nge-diazepam.

Ngomhla we-2 ku-Epreli (Usuku lwe-10): Uqalise unyango lwe-methylprednisolone kwiintsuku ze-3.

- Impendulo ye-CRS: iBakala 3, i-CRES: iBakala 3.

- ngo-Epreli 8 (Umhla we-16): Uvavanyo lomongo wethambo lubonisa ukuxolelwa okupheleleyo kwe-morphological, ukuhamba kwe-cytometry engalunganga kwimibhobho eyingozi; I-MLL-ENL ye-fusion yofuzo yohlalutyo lobungakanani: 0.