Real-world clinical outcomes of autologous stem cell transplantation in Chinese patients with newly diagnosed multiple myeloma: a systematic literature review

Abstract

Objective:

This systematic literature review analyzed real-world evidence on autologous stem cell transplantation (ASCT) for newly diagnosed multiple myeloma (ndMM) in China.

Methods:

Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, major English and Chinese databases were searched for observational studies published between 2015 and 2025 that included Chinese ndMM patients eligible for ASCT. Extracted data covered patient characteristics, treatment responses, and survival outcomes associated with ASCT and non-ASCT treatments. Single-arm meta-analysis with a random-effects model was used for evidence synthesis and pooled outcomes of the treatments for the same phase were compared using Cochran’s Q or chi-square tests.

Results:

Eighty-eight observational studies (2015–2025) were included. Data were pooled using single-arm meta-analysis and the pooled outcomes for ASCT and non-ASCT treatments were compared using Cochran’s Q or chi-square tests. Compared to patients receiving non-ASCT treatments, ASCT recipients were younger (54.5 years vs 61.4 years), had more early-stage disease (International Staging System (ISS) I: 25.8% vs 15.1%), more t(11;14) (14.1% vs 6.0%), and fewer complex karyotypes (2.9% vs 31.7%). Plerixafor plus granulocyte colony-stimulating factor (G-CSF) significantly improved mobilization success over cyclophosphamide plus G-CSF (73.6% vs 49.5%), though with slightly delayed engraftment. ASCT was associated with superior treatment response (complete response: 78.5% vs 45.3%; very good partial response (⩾VGPR): 98.6% vs 75.0%) and higher 5-year overall survival rate (70.6% vs 41.3%) and 5-year progression-free survival rate (41.3% vs 18.0%). Advanced revised-ISS stage, IgD subtype, and suboptimal pre-ASCT response were associated with poorer survival prognosis in ASCT recipients.

Conclusion:

Chinese ASCT recipients had more favorable baseline profiles and achieved significantly better clinical outcomes than those receiving non-ASCT treatments. Plerixafor-based mobilization enhanced stem cell collection success. However, advanced stage, IgD subtype, and inadequate pre-ASCT response could discount the effectiveness of ASCT.

INPLASY registration number:

INPLASY2025110069.

Plain language summary

A systematic review of real-world outcomes of autologous stem cell transplantation in Chinese patients with newly diagnosed multiple myeloma

Why was the study done? Multiple myeloma is a type of blood cancer. A treatment called autologous stem cell transplantation (ASCT) can improve outcomes, but not all patients are eligible. In China, more studies have recently looked at how ASCT works in real-world patients. This systematic review brought together results from many studies to better understand whether ASCT provides better results compared to other treatments. What did the researchers do? The research team systematically reviewed and combined results from 88 real-world studies published between 2015 and 2025. They compared patient characteristics, treatment response, and survival outcomes between patients who received ASCT and those who did not. They also looked at which factors influenced the success of ASCT. What did the researchers find? Patients who underwent ASCT were generally younger and had less advanced disease than those who did not. Using plerixafor plus G-CSF helped doctors collect stem cells more successfully than older methods. ASCT led to much higher treatment response rates and longer survival: about 7 in 10 patients were still alive 5 years after transplant, compared with about 4 in 10 without transplant. However, patients with advanced disease stage, IgD subtype myeloma, or weaker treatment response before transplant had worse outcomes even with ASCT. What do the findings mean? This systematic review shows that in real-world Chinese patients, ASCT provided much better outcomes than non-transplant treatments. Newer ways to collect stem cells, such as plerixafor-based regimens, improved success rates. However, patients with certain high-risk features may need additional treatment strategies to maximize the benefits of ASCT.

Keywords

autologous stem cell transplantation China meta-analysis newly diagnosed multiple myeloma systematic review

Introduction

Multiple myeloma (MM) is a malignant clonal plasma cell disorder and the second most common hematologic malignancy worldwide, primarily affecting older adults. Despite therapeutic advances, MM remains incurable.¹ In China, the standardized incidence was 1.15 per 100,000,² with a rising disease burden (disability-adjusted life years increasing by 134% from 1990 to 2019).³

Autologous stem cell transplantation (ASCT), introduced in the 1980s, has significantly improved overall survival (OS) and remains a cornerstone of frontline treatment for transplant-eligible patients with newly diagnosed MM (ndMM).^4,5 However, its utilization in China remains low, with only 11.3% of patients undergoing ASCT, and the highest uptake (33.8%) observed among those aged 45–49.⁶ Despite the rise of newer therapies—including proteasome inhibitors (PI), immunomodulatory drugs (IMiD), monoclonal antibodies, and chimeric antigen receptor T-cell (CAR-T) therapies—ASCT continues to offer distinct clinical advantages, providing deeper and more durable responses, longer progression-free survival (PFS), and standardized protocols supported by decades of clinical experience.^7–9 It is also more cost-effective, particularly in resource-limited healthcare systems like China.¹⁰ However, ASCT is invasive, requires hospitalization and intensive support, and may not be suitable for frail or elderly patients. In contrast, novel therapies are less invasive and better tolerated but often expensive, less accessible, and lack long-term real-world data.

To better guide the use of ASCT and maximize its clinical benefits in Chinese ndMM patients, this study aimed to leverage existing real-world evidence (RWE) to evaluate ASCT’s clinical impact, characterize its real-world utilization, and generate actionable insights to inform future research and clinical decision-making.

Methods

This study was conducted as a systematic literature review (SLR) to summarize RWE evaluating ASCT or non-ASCT treatments in Chinese patients with transplant-eligible ndMM. This SLR followed the latest guidelines outlined in the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement¹¹ to ensure methodological rigor and transparency. This study was registered poststudy at INPLASY (https://inplasy.com/): registration number INPLASY2025110069.

Data sources and search strategy

A comprehensive search strategy was applied across six major databases, including three English-language sources (MEDLINE, Embase, and Web of Science) and three Chinese-language databases (CNKI, WANFANG, and VIP). The search covered publications from January 1, 2015 to January 15, 2025. Keywords and database-specific subject headings were combined using Boolean operators, targeting four major domains: disease-related terms (“multiple myeloma,” “Kahler disease,” “plasma cell myeloma”), diagnosis phase descriptors (“newly diagnosed,” “untreated,” “first-line,” “primary,” “de novo”), geographic filters (“China,” “Chinese”), and RWE methodologies (“observational,” “prospective,” “retrospective,” “real-world,” “cohort,” “registry-based”). The full literature search strategies for all databases are presented in the Supplemental material.

Study selection: Inclusion and exclusion criteria

The search results of the included bibliographic databases were pooled to remove duplicates. Two independent reviewers screened titles and abstracts, followed by full-text review of potentially relevant articles, with any discrepancies resolved through discussion and, if needed, adjudicated by the study investigator. Eligible studies were observational (prospective, retrospective, or cross-sectional) and focused on ASCT or non-ASCT treatments for transplant-eligible ndMM in Chinese patients. Exclusion criteria included clinical trials (phases I–III), preclinical studies, case reports/series, nonhuman studies, clinical guidelines, reviews, and studies involving mixed or relapsed/refractory cohorts.

Data extraction

Data extraction was performed using structured Excel-based forms capturing five major domains: (1) study characteristics (design, setting, and data sources); (2) patient profiles (demographics, clinical staging including Durie–Salmon and International Staging System (ISS), monoclonal protein, cytogenetic features, and comorbidities); (3) treatment protocols (detailed therapeutic regimens and treatment phases); (4) clinical outcomes including treatment response across different treatment phases, ASCT-related outcomes (mobilization, engraftment times, and hematologic recovery), and survival outcomes (PFS and OS); and (5) safety outcomes (adverse event types, incidence, and Common Terminology Criteria for Adverse Events (CTCAE) grading).

Evidence synthesis and statistical analysis

Studies were categorized by clinical pathways and outcomes associated with ASCT (induction therapy, stem cell mobilization, posttransplant engraftment, and maintenance therapy) and non-ASCT treatments (induction therapy and maintenance therapy) for evidence synthesis. Data harmonization was performed to ensure consistency across outcomes. When only medians and interquartile ranges were reported, they were converted to means and standard deviations using validated statistical methods. Single-arm meta-analysis with a random-effects model was used to pool results from studies with comparable patient populations, treatment modalities, and outcome measures. Between-study heterogeneity was evaluated using the I² statistic, and subgroup analyses were performed when I² was greater than 50% to explore potential sources of heterogeneity. When meta-analysis was not feasible, findings were summarized descriptively. The pooled outcomes associated with treatments for the same outcomes were compared using Cochran’s Q test for continuous outcomes and chi-square tests for categorical outcomes. Additionally, the hazard ratios (HR) and 95% confidence intervals (CI) for the same prognostic variables from the reported Cox-regression analyses for OS associated with ASCT in the included studies were pooled using meta-analytic methods as well. Forest plot was used to illustrate the results of meta-analyses. All statistical analyses were performed using R software (version 4.2.0; R Foundation for Statistical Computing, Vienna, Austria) and statistical significance was defined as two-sided p value less than 0.05.

Results

The literature search process is illustrated in Figure 1. A total of 527 English and 592 Chinese references were identified through the initial searches. After screening titles and abstracts to exclude irrelevant studies, 159 English and 288 Chinese references were selected for full-text review. Ultimately, 88 references (37 English and 51 Chinese) met the predefined eligibility criteria and were retained for data extraction and evidence synthesis.

Figure 1.

The flowchart of literature search.

Patient characteristics of Chinese patients with transplant-eligible ndMM

The included 43 studies involving 6669 patients with transplant-eligible ndMM reported patient characteristics that enabled this SLR to compare pooled baseline characteristics stratified by treatment type: ASCT (30 studies, n = 3957^12–41) and non-ASCT treatments (19 studies, n = 2712^{12,17,18,25,27,34,42–54}). Compared with patients receiving non-ASCT treatments, ASCT recipients were significantly younger (mean age: 54.5 vs 61.4 years, p < 0.001), more likely to have IgG monoclonal protein (50.8% vs 44.9%, p < 0.001), and present with ISS stage I disease (25.8% vs 15.1%, p < 0.001). In addition, ASCT recipients had higher proportions of patients with kappa light chains (47.9% vs 13.7%, p = 0.004), lambda light chains (49.3% vs 16.5%, p < 0.001), the t(11;14) translocation (14.1% vs 6.0%, p < 0.001), and a lower proportion of complex karyotypes (2.9% vs 31.7%, p = 0.020). Detailed comparisons are presented in Table 1.

Table 1.

Pooled patient characteristics of Chinese patients receiving ASCT and non-ASCT treatments for transplant-eligible ndMM.

Variable	ASCT group			Non-ASCT group			p Value
Variable	Number of studies	Sample size	Point estimation (95% CI)	Number of studies	Sample size	Point estimation (95% CI)	p Value
Demographics
Age	28	3692	54.5% (53.2, 55.8)	18	2566	61.4% (59.4, 63.4)	<0.001
Male gender	28	3692	58.7% (56.3, 61.0)	18	2566	56.4% (54.5, 58.3)	0.152
Durie–Salmon stage
I	11	1589	5.9% (2.9, 11.5)	5	384	5.1% (1.6, 15.3)	0.825
II	12	1631	16.3% (12.3, 21.1)	5	384	11.7% (8.9, 15.3)	0.096
III	17	2332	78.5% (69.4, 85.4)	7	724	79.7% (68.8, 87.5)	0.849
IIIA	3	185	54.6% (47.4, 61.6)	2	247	64.4% (58.2, 70.1	0.040
IIIB	3	185	19.5% (14.4, 25.8)	2	247	20.3% (12.5, 31.2)	0.879
ISS stage
I	20	2549	25.8% (22.3, 29.7)	11	1,706	15.1% (11.9, 19.0)	<0.001
II	20	2549	36.2% (33.1, 39.4)	11	1,706	33.7% (30.3, 37.4	0.302
III	23	3209	35.3% (33.1, 37.6)	15	2,119	45.6% (41.6, 49.7)	<0.001
R-ISS stage
I	8	1058	17.7% (15.0, 20.7)	4	1,000	8.8% (3.9, 18.5)	0.074
II	8	1058	54.8% (47.5, 62.0)	4	1,000	57.1% (49.9, 64.1)	0.665
III	10	1578	21.8% (16.4, 28.4)	5	1,140	17.8% (12.4, 24.9)	0.365
Monoclonal protein
IgG	23	3230	50.8% (49.1, 52.6)	14	2,108	44.9% (42.4, 47.4)	<0.001
IgA	23	3230	21.1% (19.7, 22.5)	13	2,028	24.9% (22.4, 27.5)	0.008
IgD	18	2897	5.5% (4.3, 7.0)	11	1,688	5.3% (3.6, 7.7)	0.883
IgM	3	235	1.3% (0.4, 3.9)	1	104	1.0% (0.1, 6.5)	0.805
Light chain	17	2393	22.0% (19.7, 24.3)	12	1,992	22.3% (19.5, 25.4)	0.851
Nonsecretor	12	1572	3.4% (2.4, 4.7)	6	741	2.5% (1.0, 6.0)	0.513
Kappa light chain	7	946	13.7% (4.8, 33.5)	1	140	47.9% (39.7, 56.1)	0.004
Lambda light chain	7	946	16.5% (8.4, 29.8)	1	140	49.3% (41.1, 57.5)	<0.001
Cytogenetics
High risk	9	970	26.6% (15.3, 42.1)	6	1,046	24.3% (15.5, 36.0)	0.787
t(4;14)	8	1917	13.4% (9.5, 18.6)	3	722	10.4% (7.2, 14.8)	0.306
t(14;16)	7	1840	3.5% (2.7, 4.4)	2	644	2.1% (1.1, 4.1)	0.170
del(17p)	7	1439	14.2% (12.5, 16.1)	1	268	12.3% (8.9, 16.8)	0.402
Standard risk	7	413	45.0% (31.1, 59.8)	3	475	45.7% (21.8, 71.9)	0.965
t(11;14)	7	1840	14.1% (12.6, 15.8)	1	335	6.0% (3.9, 9.1)	<0.001
Complex karyotype	1	35	2.9% (0.4, 17.7)	2	295	31.7% (12.4, 60.3)	0.020
Comorbidities
Extramedullary diseases	5	582	21.3% (16.8, 26.7)	4	681	18.4% (12.8, 25.9)	0.499
Hypertension	1	93	32.3% (23.6, 42.4)	3	481	38.5% (33.0, 44.3)	0.283
Diabetes	1	93	18.3% (11.7, 27.5)	2	382	10.8% (6.7, 16.9)	0.102

ASCT, autologous stem cell transplantation; ISS, International Staging System; ndMM, newly diagnosed MM; R-ISS, Revised International Staging System.

Italic p values indicate statistical significance (p < 0.05).

Outcomes of mobilization, engraftment, and hematopoietic recovery in the ASCT receipts

Three studies (n = 212)^19,28,38 evaluated the combination of plerixafor and granulocyte colony-stimulating factor (G-CSF), while six studies (n = 419)^{13,19,20,29,39,41} assessed cyclophosphamide plus G-CSF for stem cell mobilization. Plerixafor-G-CSF demonstrated significantly higher mobilization success rate compared to cyclophosphamide-G-CSF (73.6% (I² = 59.8%) vs 49.5% (I² = 38.3%), p = 0.001), but was associated with modest delays in engraftment for neutrophils (11.0 days vs 10.5 days (I² = 78.5%), p < 0.001) and platelets (13.0 days vs 11.3 days (I² = 79.1%), p < 0.001). Heterogeneity (I²) was not reported for the plerixafor-G-CSF group because these engraftment outcomes were each derived from a single study.

Pooled analysis from 13 studies (n = 1044)^{13,18,20,21,23,24,26,28,31,35,39,41,55} reported a mean time to granulocyte engraftment (defined as an absolute neutrophil count ⩾0.5 × 10⁹/L) was 10.8 days (95% CI: 10.1–11.6, I² = 99.3%), while the mean time to platelet engraftment (defined as platelet count ⩾20 × 10⁹/L without transfusion support) was 12.2 days (95% CI: 11.0–13.4, I² = 97.4%). Subgroup analyses could only be conducted based on mobilization regimen, and all subgroups still exhibited substantial heterogeneity with I² values exceeding 90%. Additionally, pooled data from four studies (n = 327)^22,28,56,57 showed a mean time to earlier granulocyte recovery of 10.4 days and a mean time to megakaryocyte lineage recovery of 11.3 days. Details of mobilization, engraftment, and engraftment outcomes are summarized in Table 2.

Table 2.

Comparisons of pooled stem cell mobilization success and engraftment outcomes between two mobilization regimens in Chinese patients receiving ASCT for ndMM.

Variable	PXF + G-CSF			I² (%)	Cy + G-CSF			I² (%)	p Value
Variable	Number of studies	Sample size	Point estimation (95% CI)	I² (%)	Number of studies	Sample size	Point estimation (95% CI)	I² (%)	p Value
Mobilization success rate	2	87	73.6% (63.3, 81.8)	59.8	3	95	49.5% (39.6, 59.4)	38.3	0.001
CD34⁺ cells (×10⁶/kg)
After first collection	1	18	5.1 (3.6, 6.5)	—	2	65	2.3 (0, 4.8)	78.7	0.063
After third collection	1	18	7.8 (3.6, 12.0)	—	2	53	5.0 (2.1, 7.9)	0.0	0.281
MNC number (×10⁸/kg)
After first collection	1	18	4.7 (1.9, 7.6)	—	2	65	2.3 (0, 4.6)	32.3	0.189
After third collection	1	18	8.6 (2.9, 14.2)	—	1	23	3.4 (0, 8.8)	—	0.194
Granulocyte engraftment time (days)	1	125	11.0 (10.1, 11.9)	—	3	324	10.5 (9.9, 11.0)	78.5	<0.001
Platelet engraftment time (days)	1	125	13.0 (11.6, 14.4)	—	3	324	11.3 (10.7, 12.0)	79.1	<0.001

ASCT, autologous stem cell transplantation; Cy, cyclophosphamide; G-CSF, granulocyte colony-stimulating factor; MNC, mononuclear cell; ndMM, newly diagnosed MM; PXF, plerixafor.

Italic p values indicate statistical significance (p < 0.05).

Treatment response and survival outcomes of ASCT and non-ASCT treatments

The comparisons of pooled treatment response outcomes revealed that ASCT recipients achieved significantly superior effectiveness compared to those managed with non-ASCT treatments. Specifically, for stringent complete response (sCR) or complete response (CR), ASCT achieved a pooled response rate of 78.5% (seven studies, n = 526^{17,18,25,34,58–60}), which was significantly higher than the 45.3% observed for non-ASCT treatments (six studies, n = 651^{17,18,25,54,59,60}). Similarly, the pooled proportion of patients achieving at least a very good partial response (⩾VGPR) was significantly higher at 98.6% (three studies, n = 121^25,59,60) compared to 75.0% (p = 0.011) in the patients receiving non-ASCT treatments (three studies, n = 115^25,59,60; Figure 2).

Figure 2.

Comparison of pooled treatment response outcomes between ASCT and non-ASCT treatments in Chinese patients with transplant-eligible ndMM. (A) Pooled CR or better rates comparing ASCT with non-ASCT treatments. (B) Pooled VGPR or better rates comparing ASCT with non-ASCT treatments.

In terms of survival outcomes, ASCT was associated with markedly improved OS and PFS. Pooled data from 52 studies^{12–18,21–24,26,30–37,39–41,58,59,61–87} involving 5210 patients receiving ASCT showed a significantly higher 5-year OS rate of 70.6% in ASCT receipts compared to 41.3% (p < 0.001) in patients receiving non-ASCT treatments (38 studies with 5714 patients^{12,17,18,27,30,34,42,44–48,50,53,54,61–63,65–75,77–81,83,86–88}). Similarly, the pooled 5-year PFS rate was significantly higher in ASCT-treated patients, with 42.0% (49 studies with 4953 patients^{12,13,15–18,21–24,26,27,30–37,39–41,57–59,61,62,64,65,67–69,71–74,76,77,79,82,83,85–91}) versus 18.4% (p < 0.001) in patients receiving non-ASCT treatments (34 studies with 5182 patients^{12,17,18,27,30,34,44–49,51,53,54,59,61,62,65,67–69,71,73,74,77,79,83,86–88,90–92}).

Subgroup analyses were conducted to assess the impact of induction and maintenance therapies on ASCT outcomes (Figure 3). When IMiD-based maintenance was consistent posttransplant, patients who received combined PI and IMiD induction (one study, n = 33⁷¹) had a significantly higher 5-year OS rate (81.2% vs 61.0%; p = 0.037) than those with PI-only induction (four studies, n = 105^24,32,33,39). Additionally, PI-only induction therapy (five studies, n = 133^{24,32,33,39,64}) was associated with a significantly higher 5-year PFS rate (42.2% vs 16.5%, p = 0.005) than IMiD-only induction (one study, n = 39⁷¹).

Figure 3.

Comparisons of the pooled survival outcomes between induction treatments followed by IMiD maintenance in Chinese patients receiving ASCT for ndMM. (A) Pooled 5-year PFS rates for different induction regimens followed by IMiD maintenance. (B) Pooled 5-year OS rates for different induction regimens followed by IMiD maintenance.

Factors associated with survival outcomes in the ASCT receipts

Seven studies^{15,16,22,33,35,37,40} conducted multiple Cox-regression analysis exploring predictors for OS in patients receiving ASCT for ndMM. Four studies (n = 869)^33,35,37,40 found that ISS stage III was associated with significantly increased mortality (pooled HR: 1.55). Notably, early relapse (within 12 months) post-ASCT, reported in two studies (n = 450),^15,40 was associated with an exceptionally high risk of death (pooled HR: 18.52). Conversely, post-ASCT minimal residual disease (MRD) negativity was protective, as shown in one study with 156 patients¹⁵ (pooled HR: 0.23). A multicenter study (n = 401)¹⁶ indicated elevated mortality risk in patients with revised-ISS (R-ISS) stage III or IV versus stage I (HR ranging from 4.20 to 9.53). This study also highlighted a significantly higher risk of death associated with specific monoclonal protein subtypes—IgD versus IgG (HR = 4.96) and nonsecretory versus IgG (HR = 4.68). Eight studies^{15,16,18,22,35–37,41} assessed PFS predictors. Advanced R-ISS stages were consistently associated with increased progression risk: stage III versus I (HR: 2.37) and stage IV versus I (HR: 4.50).¹⁶ The IgD subtype also predicted poorer PFS (HR: 2.48).¹⁶ Partial response or less before ASCT increased progression risk (pooled HR: 2.44, 95% CI: 1.27–4.70) in two studies (n = 130),^18,22 while post-ASCT MRD negativity lowered it (pooled HR: 0.34) as reported by two studies (n = 235).^15,22 Achieving ⩾VGPR after ASCT also predicted better PFS (HR: 0.30).³⁵ Figure 4 summarizes pooled prognostic factors for OS and PFS.

Figure 4.

Pooled HR of prognostic factors for survival outcomes in Chinese patients receiving ASCT for ndMM. (A) Pooled HRs of prognostic factors for PFS. (B) Pooled HRs of prognostic factors for OS.

Severe AE related to ASCT

The pooled incidence of severe (Grade ⩾3) adverse events (AE) following ASCT indicated that diarrhea emerged as the most frequent high-grade AE, with a pooled incidence of 11.8% (95% CI: 5.6–23.4) reported across two studies (n = 177).^28,35 Infection was the second most common severe AE, occurring in 7.6% of patients (95% CI: 1.9–26.1) in two studies (n = 177).^12,55 Other notable high-grade AEs included gastrointestinal reactions, with a pooled incidence of 4.8% (95% CI: 1.9–11.6) reported in five studies (n = 533),^{12,18,28,35,55} and hepatic toxicity, reported at 3.9% (95% CI: 1.3–11.5) in two studies (n = 177).^12,55 Oral mucositis was observed in 4.6% of patients (95% CI: 2.8–7.7) across three studies (n = 302),^12,28,55 while general mucositis had a slightly higher pooled incidence of 5.1% (95% CI: 0.8–26.7) from two studies with (n = 129).^35,55

Discussion

This systematic review and meta-analysis provide a comprehensive synthesis of RWE on the clinical characteristics, procedural outcomes, treatment outcomes, and survival prognostic factors associated with ASCT in Chinese patients with ndMM. Our findings confirm that ASCT remains a key therapeutic strategy in China, offering superior depth of response, PFS, and OS compared to non-ASCT approaches. Importantly, the results also contextualize these benefits within China’s healthcare system and add critical comparisons to real-world data from Western countries, shedding light on shared challenges and region-specific differences that impact clinical decision-making.

Patients selected for ASCT in the included Chinese studies were younger and more likely to present with favorable disease features, such as IgG monoclonal protein, ISS stage I disease, and t(11;14) translocation. These characteristics closely mirror patterns observed in Western ASCT registries, such as the Center for International Blood and Marrow Transplant Research (CIBMTR) in the United States and the European Society for Blood and Marrow Transplantation (EBMT) database. For example, a recent CIBMTR analysis also reported a median ASCT age of ~55 years with comparable prognostic profiles.⁹³ However, China applies a more conservative age cutoff, often excluding patients over 65, while Western centers now consider ASCT in selected patients up to 70 or even 75 years of age.⁷ This suggests a potential underutilization of ASCT among older adults in China and highlights the need to develop frailty-based rather than age-based selection tools in the Chinese setting.

With respect to mobilization and engraftment, our study found that plerixafor plus G-CSF was more effective than cyclophosphamide-G-CSF in achieving successful stem cell mobilization, consistent with US real-world data (e.g., Afifi et al.⁹⁴). However, in China, access to plerixafor may be limited by reimbursement and availability, and our finding of slightly delayed engraftment with plerixafor—although clinically acceptable—warrants further cost-effectiveness analysis in this context. Standardizing mobilization strategies across Chinese centers based on both efficacy and affordability would help optimize transplant logistics and equity. In terms of treatment response, patients undergoing ASCT in China achieved a pooled CR/sCR rate of 78.5% and ⩾VGPR rate of 98.6%, substantially outperforming non-ASCT treatment groups. These response rates are comparable to those reported in large clinical trials from western countries. For example, an EBMT study involving >10,000 patients reported post-ASCT ⩾VGPR rates ranging from 75% to 90%, depending on induction regimen and cytogenetic risk. This indicates comparable procedural success despite differences in healthcare resources. Survival outcomes in this Chinese population were also consistent with international benchmarks. Our pooled 5-year OS rate of 70.6% in ASCT recipients is in line with Western real-world studies reporting 5-year OS ranging from 65% to 75% depending on age and risk status.^95–97 However, the 5-year OS in the Chinese non-ASCT group was notably lower (41.3%), which is less favorable than some Western real-world cohorts.^98,99 This discrepancy may reflect more limited access to salvage therapies (e.g., second-line PI, CAR-T, or bispecific antibodies) in China, highlighting the importance of optimizing ASCT during first-line treatment when later-line options are less available.

The analysis of induction and maintenance regimens revealed that patients receiving PI and IMiD combinations had significantly better long-term outcomes than those receiving single-class regimens. These findings echo global evidence, such as the PERSEUS¹⁰⁰ and GRIFFIN¹⁰¹ trials, which demonstrated that triplet or quadruplet combinations (e.g., VRd, Dara-VTd) are superior to doublets in inducing deep responses and extending PFS when used prior to ASCT. Yet, in the Chinese setting, real-world use of triplet induction regimens may still be suboptimal due to affordability, drug accessibility, or regional variation in practice. Policy efforts that expand access to optimal induction therapy—especially combinations of PI and IMiD—could further improve ASCT outcomes in China.

In addition to treatment outcomes, our analysis highlighted several prognostic factors that align with Western findings. Advanced ISS or R-ISS stage, early relapse (<12 months), and adverse immunoglobulin subtypes (e.g., IgD or nonsecretory) were consistently associated with worse OS and PFS, while MRD negativity and post-ASCT ⩾VGPR predicted superior outcomes. Similar patterns have been reported in CIBMTR¹⁰² and Spanish PETHEMA registry analyses,¹⁰³ reinforcing their global relevance. However, MRD assessment remains inconsistently applied in Chinese clinical practice due to limited standardization and reimbursement barriers. Expanding the use of MRD as a prognostic and therapeutic tool represents a key opportunity to advance precision care in Chinese transplant programs.

Importantly, the safety profile of ASCT in this review was comparable to Western data, with low incidence rates of grade ⩾3 AE. Diarrhea (11.8%) and infection (7.6%) were the most common toxicities, consistent with published Western ASCT cohorts.¹⁰⁴ These data support the overall tolerability of ASCT in Chinese patients at experienced centers. However, older and comorbid patients remain underrepresented in the Chinese literature, suggesting a need to develop real-world frailty-adapted transplant pathways—like those now adopted in Europe and North America.

Despite the valuable insights provided, several limitations of this review must be acknowledged. First, the included studies were predominantly retrospective, and heterogeneity in study design, patient selection, and endpoint definitions limits comparability across sources. Second, detailed data on cytogenetic subtypes, performance status, comorbidities, and socioeconomic factors were limited, restricting our ability to perform more granular subgroup analyses. Third, this study was unable to conduct adjusted comparisons of treatment outcomes between ASCT and non-ASCT treatments due to the lack of studies directly comparing the two approaches. Given that ASCT recipients were typically younger with more favorable disease profiles, the treatment effect of ASCT may be overestimated. Poststudy registration may have introduced bias to our results and is a limitation of this study. Thus, future research should focus on addressing these gaps through prospective, multicenter studies that integrate standardized assessment approaches for patient frailty, disease severity, ASCT procedural outcomes, and treatment outcomes, allowing for adjusted comparison analysis using patient-level data.

Conclusion

In conclusion, this comprehensive SLR confirms that ASCT remains a highly effective and safe standard of care for transplant-eligible Chinese patients with ndMM. When combined with modern induction regimens and structured post-ASCT monitoring, ASCT leads to high response rates and favorable survival outcomes, comparable to those observed in Western countries for ndMM. However, the observed superiority of ASCT over non-ASCT treatments in the included real-world studies reflects unadjusted outcomes reported by the original studies and could be heavily influenced by baseline patient characteristics, including the younger age and lower ISS stage of ASCT recipients. Future real-world studies using robust methods to adjust for confounding are needed to more accurately quantify the comparative effectiveness of ASCT.

Supplemental Material

sj-docx-1-tah-10.1177_20406207261417132 – Supplemental material for Real-world clinical outcomes of autologous stem cell transplantation in Chinese patients with newly diagnosed multiple myeloma: a systematic literature review

Supplemental material, sj-docx-1-tah-10.1177_20406207261417132 for Real-world clinical outcomes of autologous stem cell transplantation in Chinese patients with newly diagnosed multiple myeloma: a systematic literature review by Jiawen You, Yuanyuan Zhang, Yuan Meng, Yiming Zhao, Huiqing Huang, Wendong Chen and Jianbo Meng in Therapeutic Advances in Hematology

Supplemental Material

sj-docx-2-tah-10.1177_20406207261417132 – Supplemental material for Real-world clinical outcomes of autologous stem cell transplantation in Chinese patients with newly diagnosed multiple myeloma: a systematic literature review

Supplemental material, sj-docx-2-tah-10.1177_20406207261417132 for Real-world clinical outcomes of autologous stem cell transplantation in Chinese patients with newly diagnosed multiple myeloma: a systematic literature review by Jiawen You, Yuanyuan Zhang, Yuan Meng, Yiming Zhao, Huiqing Huang, Wendong Chen and Jianbo Meng in Therapeutic Advances in Hematology

Footnotes

Acknowledgements

None.

Declarations

ORCID iD

Jianbo Meng

Supplemental material

Supplemental material for this article is available online.

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