MIC therapy for specific immunosuppression in transplantation
11554139 · 2023-01-17
Assignee
Inventors
- Christian Morath (Heidelberg, DE)
- Anita Schmitt (Bammental, DE)
- Matthias Schaier (Heidelberg, DE)
- Gerhard Opelz (Heidelberg, DE)
- Peter Terness (Eppelheim, DE)
- Christian Kleist (Neckargemünd, DE)
- Volker Daniel (Heidelberg, DE)
- Caner Süsal (Dossenheim, DE)
- Michael Schmitt (Bammental, DE)
- Martin Zeier (Gaiberg, DE)
Cpc classification
A61K35/15
HUMAN NECESSITIES
A61K31/706
HUMAN NECESSITIES
A61K31/164
HUMAN NECESSITIES
A61P37/06
HUMAN NECESSITIES
A61K31/405
HUMAN NECESSITIES
A61K31/422
HUMAN NECESSITIES
International classification
A61K35/15
HUMAN NECESSITIES
A61K31/405
HUMAN NECESSITIES
A61K31/164
HUMAN NECESSITIES
A61P37/06
HUMAN NECESSITIES
A61K31/407
HUMAN NECESSITIES
A61K31/706
HUMAN NECESSITIES
Abstract
The present invention relates to pharmaceutical compositions with isolated and treated whole blood cells or Peripheral Blood Mononuclear Cells (PBMCs) as well as such pharmaceutical compositions for use in the prevention and/or treatment of organ or cell graft rejection in a human graft recipient.
Claims
1. A dosage of a pharmaceutical composition for a human graft recipient comprising: a) isolated whole blood cells and/or Peripheral Blood Mononuclear Cells (PBMCs) treated with a therapeutically effective amount of mitomycin C, wherein the isolated whole blood cells or PBMCs are obtained or derived from a human graft donor, and b) optionally a pharmaceutically acceptable carrier; wherein the dosage prevents or treats organ or cell graft rejection in the human graft recipient, and wherein the dosage comprises at least 1×10.sup.8 cells of the treated whole blood cells and/or the treated PBMCs.
2. The dosage according to claim 1 comprising Peripheral Blood Mononuclear Cells (PBMCs), wherein the PBMCs comprise monocytes and lymphocytes.
3. The dosage according to claim 1 comprising the pharmaceutically acceptable carrier, wherein the pharmaceutically acceptable carrier is selected from of sugars; starches; cellulose and its derivates; powdered tragacanth; malt; gelatine; talc; cocoa butter; suppository waxes; oils; glycols; esters; agar; buffering agents; alginic acid; pyrogen-free water; isotonic saline; Ringer's solution; ethyl alcohol; phosphate buffer solutions; non-toxic, compatible lubricants; colouring agents; releasing agents; coating agents; sweetening, flavouring and perfuming agents; preservatives; and antioxidants.
4. A method for producing a dosage according to claim 1 comprising: a) obtaining a blood cell sample from the human graft donor and optionally preparing Peripheral Blood Mononuclear Cells (PBMCs) from said blood cell sample, b) treating the cells obtained in a) ex vivo with a therapeutically effective amount of mitomycin C, and c) optionally, providing a pharmaceutically acceptable carrier and adding the carrier to the treated cells obtained in b).
5. The method according to claim 4, wherein the total amount of the treated whole blood cells or the treated PBMCs in the dosage is at least 1.5×10.sup.8 cells per kg body weight of the recipient.
6. The method according to claim 4, wherein the pharmaceutically acceptable carrier is present and selected from sugars; starches; cellulose and its derivatives; powdered tragacanth; malt; gelatine; talc; cocoa butter; suppository waxes; oils; glycols; agar; buffering agents; alginic acid; pyrogen-free water; isotonic saline; Ringer's solution; ethyl alcohol; phosphate buffer solutions; non-toxic, compatible lubricants; colouring agents; releasing agents; coating agents; sweetening, flavouring, and perfuming agents; preservatives; and antioxidants.
7. A method for preventing and/or treating organ and/or cell graft rejection in a human graft recipient comprising administering to the recipient an amount of a pharmaceutical composition comprising: a) isolated whole blood cells and/or Peripheral Blood Mononuclear Cells (PBMCs) treated with a therapeutically effective amount of mitomycin C, wherein the isolated whole blood cells or PBMCs are obtained or derived from a human graft donor; and b) optionally a pharmaceutically acceptable carrier; wherein the amount of the pharmaceutical composition administered to the recipient comprises at least 1×10.sup.8 cells of the treated whole blood cells and/or the treated PBMCs per kg body weight of the recipient; wherein the pharmaceutical composition is administered to the recipient at least five days before an organ and/or cell transplantation.
8. The method according to claim 7, comprising administering to the recipient at least 1.5×10.sup.8 cells of the treated whole blood cells or the treated PBMCs per kg body weight of the recipient.
9. The method according to claim 7, wherein the administration is performed as an intravenous, intramuscular, or subcutaneous injection, or as an infusion.
10. The method according to claim 7 comprising administering to the recipient at least 1.5×10.sup.8 cells of the treated whole blood cells or the treated PBMCs per kg body weight of the recipient, wherein the administration is performed at least 7 days before the transplantation.
Description
DESCRIPTION OF THE FIGURES
(1)
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(9) Also described herein is a method for preventing or treating organ graft rejection or graft-versus-host disease in a human graft recipient, said method comprising the following steps: a) obtaining a blood cell sample from the donor, preferably from the graft donor, and optionally preparing Peripheral Blood Mononuclear Cells (PBMCs) from said blood cell sample, b) treating said blood cell sample or PBMCs derived therefrom with a therapeutically effective amount of an active substance, and c) administering the treated blood cell sample or PBMCs derived therefrom to the graft recipient and thereby treating an organ or cell graft rejection,
(10) wherein the amount of the cells of the administered treated blood cell sample or PBMCs derived therefrom is at least 0.25×10.sup.6 cells per kg body weight, preferably at least 1.5×10.sup.6 cells per kg body weight of the recipient.
(11) All the features described with regard to the pharmaceutical composition and/or the pharmaceutical composition for use also apply for the method described herein, where applicable.
(12) For this method it is preferred that the treated blood cells or PBMCs administered to the recipient comprise or consist of lymphocytes, monocytes and/or dendritic cells.
(13) Further preferred in this method is that the chemotherapeutic agent is selected from the group consisting of mitomycin C, C2 ceramide, tunicamycin, mycophenolate-mofetil, tryptophan metabolites and semisynthetic derivatives thereof, and proteasome inhibitors.
(14) It is also preferred in the method that the active substance comprises or consists of tryptophan metabolites (such as kynurenines, e.g. Tranilast) and/or mitomycin C.
(15) Preferably in this method, the amount of the cells of the administered treated blood cell sample or PBMCs derived therefrom is at least 5×10.sup.6 cells, at least 1×10.sup.7 cells, at least 5×10.sup.7 cells, at least 1×10.sup.8 cells or at least 1.5×10.sup.8 cells per kg body weight of the recipient.
(16) Further preferred for this method is that the treated blood cell sample or PBMCs derived therefrom is/are administered to the recipient at least 1 day before the transplantation, at least 2 days, at least 3 days, at least 4 days, at least 5 days, at least 6 days, at least 7 days, at least 8 days, at least 9 days, at least 10 days, at least 14 days, at least 21 days or at least 28 days before the transplantation.
(17) The skilled person will be well aware of the fact that the specific combination of cell number and time of application may depend on several individual factors of the subject such as the severity of the disorder, the age, body weight, general health, sex and diet of the patient, route of administration, the duration of the treatment as well as drugs used in combination or coincidental with the treatment or may depend on the organ to be transplanted.
(18) Preferably in this method, the administration of the treated blood cell sample or PBMCs derived therefrom is performed as intravenous injection, preferably as infusion.
(19) Also described herein is a further aspect relating to a method for treating or preventing organ graft rejection in a human graft recipient, wherein the method comprises administering to the recipient a composition comprising: a) isolated blood cells or Peripheral Blood Mononuclear Cells (PBMCs) treated with a therapeutically effective amount of an active substance, and b) optionally a pharmaceutically acceptable carrier,
(20) wherein the amount of the treated blood cells or PBMCs in the composition is selected such that at least 0.25×10.sup.6 cells, preferably at least 1.5×10.sup.6 cells of the treated blood cells or PBMCs per kg body weight of the recipient are administered.
(21) Preferably, the method may also be suitable for treating and/or preventing of graft versus host disease.
(22) All the features described with regard to the pharmaceutical composition and/or the pharmaceutical composition for use also apply for the method described herein, where applicable.
(23) In this method it is preferred that the treated blood cells or PBMCs administered to the recipient comprise lymphocytes, monocytes and/or dendritic cells.
(24) It is particularly preferred that the active substance comprises or consists of one or more substances selected from the group consisting of mitomycin C, C2 ceramide, tunicamycin, mycophenolate-mofetil, tryptophan metabolites (such as kynurenines, e.g. Tranilast) and semisynthetic derivatives thereof.
(25) It is also preferred in the method that the active substance comprises or consists of tryptophan metabolites (such as kynurenines, e.g. Tranilast) and/or mitomycin C.
(26) Preferably in this method, the amount of the cells of the administered treated blood cell sample or PBMCs derived therefrom is at least 5×10.sup.6 cells, at least 1×10.sup.7 cells, at least 5×10.sup.7 cells, at least 1×10.sup.8 cells or at least 1.5×10.sup.8 cells per kg body weight of the recipient.
(27) Further preferred for this method is that the treated blood cell sample or PBMCs derived therefrom is/are administered to the recipient at least 1 day before the transplantation, at least 2 days, at least 3 days, at least 4 days, at least 5 days, at least 6 days, at least 7 days, at least 8 days, at least 9 days, at least 10 days, at least 14 days, at least 21 days or at least 28 days before the transplantation.
(28) The skilled person will be well aware of the fact that the specific combination of cell number and time of application may depend on several individual factors of the subject such as the severity of the disorder, the age, body weight, general health, sex and diet of the patient, route of administration, the duration of the treatment as well as drugs used in combination or coincidental with the treatment or may depend on the organ to be transplanted.
(29) Preferably in this method, the administration of the treated blood cell sample or PBMCs derived therefrom is performed as intravenous injection, preferably as infusion.
(30) Preferred embodiments and further aspects of the present invention emerge from the attached claims and the following examples, the examples not being intended to limit the invention.
EXAMPLES
Example 1
TOL-1 Study
(31) A 30-day single-arm, single-centre phase I clinical trial for the determination of safety and feasibility of the intravenous administration of MICs for individualized immunosuppression was carried out in living donor kidney transplant recipients (TOL-1 Study, Ethics number: AFmo-549/2014, EudraCT number: 2014-002086-30, ClinicalTrials.gov Identifier: NCT02560220) followed by an observational phase up to day 360 after transplantation (Ethics numbers: 082/2005, 083/2005, S-395/2011). The study was performed in compliance with the provisions of the Declaration of Helsinki and the Good Clinical Practice Guidelines.
(32) Primary outcome measure was the safety and feasibility of intravenous administration of MICs as measured by the frequency of adverse events (AEs) in patients with chronic kidney disease stage 4 or 5 (i.e. GFR <30 mL/min) who received a kidney transplant from a living donor. AEs were recorded according to Common Terminology Criteria for Adverse Events (CTCAE), version 4.03.
(33) From August 2015 to February 2017, 14 donor and recipient pairs were screened for inclusion into the study. A total of 12 donors received leukapheresis and finally 10 patients were treated with the MIC product. The observation phase was for an additional 330 days up to February 2018.
(34) 10 patients (groups A, B, C) received MICs from their donor prior to kidney transplantation. Patients received either 1.5×10.sup.6 MICs per kg body weight on day −2 (N=3, group A) or 1.5×10.sup.8 MICs per kg body weight on day −2 (N=3, group B) or day −7 (N=4, group C) prior to living donor kidney transplantation.
(35) Baseline characteristics of patients are given in Table 1.
(36) TABLE-US-00001 TABLE 1 Baseline patient characteristics Total Screened treated Group A Group B Group C (N = 14) (N = 10) (N = 3) (N = 3) (N = 4) Patient Age (years) - median (range) 45 (22-59) 40 (22-59) 36 (34-47) 28 (22-59) 46 (29-50) Male sex - N (%) 11 (79) 8 (80) 3 (100) 2 (67) 3 (75) Cause of ESRD - N (%) Vascular 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) Diabetes mellitus 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) Glomerulonephritis 8 (57) 5 (50) 2 (67) 1 (33) 2 (50) Polycystic kidney disease 2 (14) 2 (20) 0 (0) 1 (33) 1 (25) Other 4 (29) 3 (30) 1 (33) 1 (33) 1 (25) Living donor Living related - N (%) 10 (71) 8 (80) 2 (67) 2 (67) 4 (100) Age (years) - median (range) 54 (42-68) 54 (42-61) 51 (46-58) 54 (47-61) 55 (42-57) Male sex - N (%) 4 (29) 3 (30) 0 (0) 1 (33) 2 (50) Serological data CMV serologic status - N (%) Donor negative, recipient negative 5 (36) 3 (30) 1 (33) 1 (33) 1 (25) Donor negative, recipient positive 1 (7) 0 (0) 0 (0) 0 (0) 0 (0) Donor positive, recipient positive 5 (36) 5 (50) 1 (33) 2 (67) 2 (50) Donor positive, recipient negative 3 (21) 2 (20) 1 (33) 0 (0) 1 (25) EBV virus serologic status - N (%) Donor negative, recipient negative 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) Donor negative, recipient positive 1 (7) 0 (0) 0 (0) 0 (0) 0 (0) Donor positive, recipient positive 12 (86) 10 (100) 3 (100) 3 (100) 4 (100) Donor positive, recipient negative 1 (7) 0 (0) 0 (0) 0 (0) 0 (0) Immunological data HLA A-, B-, DR-mismatches - N (%) 0 2 (14) 2 (20) 1 (33) 0 (0) 1 (25) 1 1 (7) 1 (10) 0 (0) 0 (0) 1 (25) 2 1 (7) 1 (10) 0 (0) 0 (0) 1 (25) 3 6 (43) 4 (40) 1 (33) 2 (67) 1 (25) 4 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) 5 4 (29) 2 (20) 1 (33) 1 (33) 0 (0) 6 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) Patients with sensitizing events - N (%) Transplantation 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) Blood transfusion 3 (21) 2 (20) 0 (0) 0 (0) 2 (50) Pregnancy 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) PRA (%) - median (range) T cell (−DTT) 0 (0-6) 0 (0-6) 0 (0-0) 0 (0-1) 2 (0-6) T cell (+DTT) 0 (0-4) 0 (0-4) 0 (0-0) 0 (0-0) 2 (0-4) B cell (−DTT) 0 (0-12) 0 (0-12) 0 (0-0) 0 (0-12) 0 (0-4) B cell (+DTT) 0 (0-35) 2 (0-35) 4 (0-12) 0 (0-35) 2 (0-4) Luminex (HLA class I) 0 (0-2) 0 (0-2) 0 (0-0) 1 (0-2) 0 (0-2) Luminex (HLA class II) 0 (0-9) 1 (0-9) 0 (0-1) 0 (0-2) 3 (0-9) Patients with pretransplant DSA - N (%) 0 (0) 0 (0) 0 (0) 0 (0) 0 (0) CMV = cytomegalovirus, DSA = donor-specific HLA A-, B-, DR-, DQ-antibodies, DTT = dithiothreitol, EBV = Epstein-Barr virus, ESRD = end-stage renal disease, HLA = human leukocyte antigen, PRA = panel-reactive antibody
(37) Four patients (R8, R9, R10, R13) were screened but received no cell therapy.
(38) All other patients (N=10: R1, R2, R3, R4, R5, R6, R7, R11, R12, R14) as well as the corresponding donors were treated per protocol (as described above).
(39) Unstimulated donor leukapheresis was performed with a Spectra Optia® apheresis device (Terumo BCT, Eschborn, Germany). MICs were produced under Good Manufacturing Practice (GMP) conditions. The MIC product was administered to the patients at the same day of donor leukapheresis and product preparation as a one-time administration. Patients of group A had a prescribed dose of 1.5×10.sup.6 MICs per kg b.w. 2 days before transplantation. Patients of group B had a prescribed dose of 1.5×10.sup.8 MICs per kg b.w. 2 days before transplantation. In patients of group C, MICs were administered at a prescribed dose of 1.5×10.sup.8 MICs per kg b.w. already 7 days prior to transplantation. The patients were assigned to the groups A (R1-R3), B (R4-R6) or C (R7, R11, R12, R14) and received the MICs. The actual administered MIC doses are given in Table 2.
(40) TABLE-US-00002 TABLE 2 Unstimulated leukapheresis in 12 donors GMP Clinical parameter Absolut Quality control of MIC end-product Production Blood Blood Achieved MIC nuclear cell CD14.sup.+ Endotoxin Mitomycin C Donor Recipient group group cell dose end-product amount ×10.sup.8 cells Sterility (EU/mL, (μg/mL, Group no. no. donor recipient per kg b.w. volume (mL) (Neubauer) (%) (Ph.Eur.) Ph.Eur.) Ph.Eur.) A D1 R1 A Rh. pos. A Rh. pos. 1.5 × 10.sup.6 85.1 1.3 11.7 sterile 0.79 <LOD A D2 R2 A Rh. pos. A Rh. pos. 1.5 × 10.sup.6 80.7 1.2 10.4 sterile 1.38 0.1 A D3 R3 O Rh. pos. O Rh. pos. 1.5 × 10.sup.6 79.1 1.2 19.2 sterile 1.56 0.0 B D4 R4 A Rh. pos. A Rh. pos. 1.5 × 10.sup.8 93.8 105.0 23.2 sterile 0.72 0.0 B D5 R5 O Rh. pos. O Rh. pos. 1.5 × 10.sup.8 85.0 87.0 17.2 sterile 1.72 0.0 B D6 R6 O Rh. pos. O Rh. neg. 0.4 × 10.sup.8* 91.6 43.0 16.2 sterile 1.31 0.0 C D7 R7 O Rh. neg. O Rh. neg. 1.5 × 10.sup.8 64.3 134.0 16.4 sterile 1.08 0.0 C D11 R11 O Rh. neg. A Rh. neg. 1.5 × 10.sup.8 91.8 96.0 15.9 sterile 1.97 0.5 C D12 R12 O Rh. pos. O Rh. pos. 1.3 × 10.sup.8# 96.7 126.0 17.3 sterile 1.39 0.3 C D14 R14 O Rh. pos. A Rh. pos. 1.5 × 10.sup.8 95.7 124.5 9.9 sterile 1.23 0.2 *In donor D6, only 60% of the targeted blood volume was processed during leukapheresis due to venous access problems and therefore patient R6 (male, 101 kg b.w ) received a reduced dose of only 0.4 × 10.sup.8 MICs per kg b.w,. #In donor D12. leukapheresis was performed per protocol but the cells obtained were only sufficient for a cell dose of 1.3 × 10.sup.8 MICs per kg b.w. in patient R12 (male, 105 kg b.w.). b.w. = body weight, CD = cluster of differentiation, EU = endotoxin units, GMP = Good Manufacturing Practice, LOD = limit of detection, MIC = modified immune cells, n.a. = not applicable, pat. = patient, Ph.Eur. = Pharmacopoea Europaea
(41) Patients were treated in a stepwise approach with a dose escalation from group A to group B to account for possible adverse reactions to the MIC end product such as embolism, inflammation, allergy or an adverse reaction to mitomycin C or the buffer. Since a too small MIC cell number confers the risk of recipient sensitization (recipients of group A had only 1% of the MIC cell number of patients of groups B and C), MICs were administered at least 2 days before transplantation during the dose escalation phase. From group B to group C, the administration time point was changed from day −2 to day −7 before transplantation.
(42) During recipient visit V3 on day −1 before transplant surgery, recipient sensitization by the MIC product was excluded by CDC and ELISA crossmatch and HLA antibody screening by the Luminex technique.
(43) Post-transplant recipient visits were on day 7±1 (V4) and day 30±4 (V5, end of study). The observation phase was up to day 360 after kidney transplantation with frequent outpatient visits on days 60±14, 90±14, 135±21, 180±21, 225±28, 270±28, 360±35.
(44) Immunosuppression consisted of cyclosporin A (CyA), enteric-coated mycophenolic sodium (EC-MPS) and methylprednisolone (MPS) given from the day of surgery. In patients of group C, immunosuppressive therapy was reduced to low dose CyA and low dose EC-MPS without steroids during the observation phase beyond day 30 to avoid infectious complications of a combined cell-based and immunosuppressive therapy. Patients of groups A and B received immunosuppression according to the standard of care. These standards are known to the skilled person. The immunosuppression applied to patients of the control group was selected such that it resulted in an immunosuppression comparable to the one obtained in patients of group C. The detailed immunosuppressive therapy in patients during the TOL-1 study and observation phase is given in
(45) Data collected during the first 30 days after transplantation showed that MIC infusions were excellently tolerated. A total of 69 adverse events (AEs) including 3 severe AEs (SAEs) occurred in the 10 treated patients. AEs were either unlikely related (N=1) or unrelated (N=68) to MIC therapy. During the study phase, no positive crossmatch results, de novo donor specific antibodies, or rejection episodes occurred with excellent kidney graft function in all patients (cf.
Example 1.1
No Detectable Donor Chimerism After MIC Infusion
(46) Already one day after MIC infusion, no donor chimerism was detectable in the 10 patients. Absence of donor chimerism was confirmed on day −1 before, and on days 7 and 30 after transplantation.
Example 1.2
Excellent Clinical Outcomes Up to One Year After Transplantation
(47) During the observation phase, no de novo donor specific antibodies, or rejection episodes occurred and all patients had an excellent kidney graft function (cf.
(48) All patients showed excellent kidney graft function with no proteinuria up to day 360 after surgery (cf.
(49) TABLE-US-00003 TABLE 3 Outcomes and complications in 10 patients (TOL-1 study phase and observation phase up to day 360) Total treated Group A Group B Group C (N = 10) (N = 3) (N = 3) (N = 4) Biopsy-proven rejection (≥BANFF IA) - N (%) 0 (0) 0 (0) 0 (0) 0 (0) Development of de novo DSA (A, B, DR, DQ) at a 0 (0) 0 (0) 0 (0) 0 (0) cut-off of >1,000 MFI - N (%) Patients with opportunistic infections - N (%) 0 (0) 0 (0) 0 (0) 0 (0) Infectious episodes - N 0 0 0 0 Pneumonia 0 0 0 0 CMV reactivation >1,000 copies/mL 0 0 0 0 BKV replication >1,000 copies/mL 0 0 0 0 BKV-associated nephropathy 0 0 0 0 Other infection 0 0 0 0 Patients with non-opportunistic infections - N (%) 4 (40) 1 (33) 0 (0) 3 (75) Infectious episodes - N 10 1 0 9 CV-associated infection 1 1 0 0 Urinary tract infection 6 0 0 6 Post-operative wound infection 1 0 0 1 Pneumonia 1 0 0 1 Other infection 1 0 0 1 PTLD or malignancy - N (%) 0 (0) 0 (0) 0 (0) 0 (0) Blood pressure on day 360 - median (range) Systolic blood pressure (mmHg) 125 (110-145) 130 (110-145) 120 (120-130) 129 (120-140) Total anti-hypertensive TIS 0.75 (0.13-2.5) 0.75 (0.5-1.5) 1.5 (0.13-1.5) 0.75 (0.17-2.5) Change of total anti-hypertensive TIS from baseline −0.22 (−4.63-0.75) −0.25 (−2.25-0) 0 (−0.19-0.75) −1.04 (−4.63-0) NODAT - N (%) 0 (0) 0 (0) 0 (0) 0 (0) Leukopenia <3.5/nL - N (%) 0 (0) 0 (0) 0 (0) 0 (0) Documented diarrhea - N (%) 0 (0) 0 (0) 0 (0) 0 (0) Patients with surgical complications* - N (%) 4 (40) 0 (0) 2 (67) 2 (50) Bleeding 1 (10) 0 (0) 0 (0) 1 (25) Wound healing disturbances 2 (20) 0 (0) 2 (67) 0 (0) Urinary leakage 1 (10) 0 (0) 0 (0) 1 (25) Lymphocele 0 (0) 0 (0) 0 (0) 0 (0) *requiring intervention. BKV = BK virus, CMV = cytomegalovirus, CV = central venous catheter, DGF = delayed graft function, DSA = donor-specific antibody, MFI = mean fluorescence intensity, NODAT = new-onset diabetes after transplantation, PTLD = post-transplant lymphoproliferative disease, TIS = therapeutic intensity score
(50) Additionally, protocol biopsies on day 7 after transplantation showed no allograft rejection. Indication biopsies due to a transient rise in serum creatinine were performed in patients R2 and R14 but showed no abnormalities. In both patients, serum creatinine returned to baseline without further measures. Patient R1 from group A, who received only 1% of the MIC cell number administered to groups B and C, was found to have borderline changes in a biopsy on day 77. In this patient, the biopsy was performed after a rise in serum creatinine of 0.2 mg/dL from baseline. Patient R11 was found to have E. coli urinary tract infection on day 128 after surgery requiring a quinolone antibiotic. Serum creatinine rose from 1.31 mg/dL before antibiotic treatment to a maximum of 1.81 mg/dL on day 157. A biopsy procedure revealed severe interstitial inflammation (i3) with nearly absent tubulitis (t1) indicative for allergic interstitial nephritis. Methylprednisolone was given at 125 mg per 3 days and serum creatinine returned to baseline in both patients.
(51) Therefore, it was followed that also the biopsies revealed that no allograft rejection occurred.
Example 1.3
Anti-Donor T Lymphocyte Responses in MIC-Treated Patients
(52) Patients from group C showed preserved lymphocyte proliferation in response to unspecific polyclonal stimulators on day 360 after transplantation as compared to before MIC infusion, indicating an intact general immune response (cf.
Example 1.4
HLA Antibodies and Antibody Titers to Bacterial and Viral Immunizations
(53) During the observation phase no de novo donor-specific antibodies were detected. This finding raised the question whether the memory B cell response to donor-unrelated antigens as induced by previous immunizations was also affected. Titer for measles (median 4400 mIU/mL, 200-11,000), mumps (median 400, 230-8000), rubella (median 41 IU/mL, 9-160), varicella (median 1350 mIU/mL, 410-3500), diphtheria (median 0.165 IU/mL, 0.04-0.33) and tetanus (median 1.45 IU/mL, 0.5-2.1) were lowest on day 30 after kidney transplantation, but reached pre-transplant levels during the further course.
Example 1.5
Pre-Transplant Levels of T Lymphocyte-, B Lymphocyte- and NK Cell Numbers One Year After Transplantation
(54) The number of CD4.sup.+ and CD8.sup.+ T lymphocytes as well as of activated CD4.sup.+ and CD8.sup.+ T lymphocytes remained stable before and after transplantation. CD19.sup.+ B lymphocytes were highest on day 30 after surgery with a median of 300/μL (149-561) but returned to pre-transplant levels on day 180 with a median of 35/μL (25-247). CD16.sup.+CD56.sup.+ NK cells behaved inversely being lowest on day 30 post-transplant with a median of 60/μL (33-73) but increased to a median of 104 (93-154) on day 180.
Example 1.6
Unchanged Number of Regulatory T Lymphocytes But Strongly Increased Number of Regulatory B Lymphocytes Starting from Day 135 After Transplantation
(55) The regulatory T lymphocyte (Treg) number was low on day 30 after transplantation (cf.
(56) Interestingly, the percentage of CD19.sup.+CD24.sup.highCD38.sup.high immature B lymphocytes (regulatory B lymphocytes, Bregs) was low until day 90 after transplantation with a median of 2.0% (0.1-5.5) on day 30 (cf.
(57) Breg percentages in patients of group C in comparison to Breg percentages of 40 measurements in 31 matched kidney transplant recipients without MIC infusion are shown in
(58) It was of particular interest, whether Bregs also increased in patients of groups A (receiving a reduced cell dose on day −2) and B (receiving the full cell dose on day −2). For this purpose, we analyzed Bregs from frozen samples. As expected, patients from group C showed the highest Breg percentages exceeding the values in patients of groups A and B by a factor of 68 and 20, respectively, on day 180 after transplantation (cf.