Desmoplastic/MBEN medulloblastoma and/or tumors with SHH signaling

In the German Hirntumor (HIT) 2000 multicenter trial, a multiagent chemotherapy regimen that included high-dose intravenous and intraventricular methotrexate was used. This drug regimen did not include high-dose chemotherapy supported by bone marrow or peripheral stem cell rescue.[81]

In the German Hirntumor (HIT) 2000 multicenter trial, a multiagent chemotherapy regimen that included high-dose intravenous and intraventricular methotrexate was used. This drug regimen did not include high-dose chemotherapy supported by bone marrow or peripheral stem cell rescue.[81]

Nineteen patients with desmoplastic medulloblastoma or MBEN had a 5-year EFS rate of 90% (±7%) and an OS rate of 100% (±0%).

In the German Hirntumor (HIT) 2000 multicenter trial, a multiagent chemotherapy regimen that included high-dose intravenous and intraventricular methotrexate was used. This drug regimen did not include high-dose chemotherapy supported by bone marrow or peripheral stem cell rescue.[81]

All patients were treated with postoperative chemotherapy alone, and no radiation was given before progression.

An expanded cohort of the German HIT 2000 trial included an additional 23 children with nodular desmoplasia or MBEN who were treated with the same regimen. The following results were reported:[111]

An expanded cohort of the German HIT 2000 trial included an additional 23 children with nodular desmoplasia or MBEN who were treated with the same regimen. The following results were reported:[111]

Combined results confirmed the excellent survival, with a 5-year EFS rate of greater than 90%.

An expanded cohort of the German HIT 2000 trial included an additional 23 children with nodular desmoplasia or MBEN who were treated with the same regimen. The following results were reported:[111]

In this expanded cohort, molecular characterization was performed and a subset of tumors with SHH signaling were identified. These patients with tumors demonstrating SHH signaling had a similar excellent prognosis.

An expanded cohort of the German HIT 2000 trial included an additional 23 children with nodular desmoplasia or MBEN who were treated with the same regimen. The following results were reported:[111]

Further characterization of the SHH signaling molecular subtype by chromosomal aberrations did not identify any differences in EFS or OS.

An expanded cohort of the German HIT 2000 trial included an additional 23 children with nodular desmoplasia or MBEN who were treated with the same regimen. The following results were reported:[111]

Other studies have suggested that further subdivision by chromosomal aberrations in young children with SHH-driven medulloblastoma was predictive of outcome.

A COG clinical trial (CCG-9921) also had a favorable outcome for children with desmoplastic medulloblastoma (including MBEN). In this study, patients with desmoplastic tumors did not receive radiation therapy before progression.[103]

A COG clinical trial (CCG-9921) also had a favorable outcome for children with desmoplastic medulloblastoma (including MBEN). In this study, patients with desmoplastic tumors did not receive radiation therapy before progression.[103]

Patients in the desmoplastic group achieved an EFS rate of 77% (±9%) and an OS rate of 85% (±8%), compared with an EFS rate of 17% (±5%) and an OS rate of 29% (±6%) for patients in the nondesmoplastic group (P < .0001 for both EFS and OS comparisons).

The COG-ACNS1221 (NCT02017964) study tested systemic chemotherapy that was identical to the chemotherapy used in the German HIT 2000 trial, except for the omission of intraventricular methotrexate.[28]

The COG-ACNS1221 (NCT02017964) study tested systemic chemotherapy that was identical to the chemotherapy used in the German HIT 2000 trial, except for the omission of intraventricular methotrexate.[28]

The study was closed early because of a higher-than-expected rate of relapse, with a 2-year PFS rate of 52% in the 25 patients who were studied.

Another treatment option for children younger than 3 years at diagnosis is high-dose chemotherapy. Results of trials using higher-dose, marrow-ablative chemotherapeutic regimens supported by stem cell rescue have also demonstrated that a subgroup of patients with medulloblastoma who are younger than 3 years and, in some studies, younger than 5 years at the time of diagnosis can be successfully treated with chemotherapy alone.[104,106,112][Level of evidence B4]; [113]

Another treatment option for children younger than 3 years at diagnosis is high-dose chemotherapy. Results of trials using higher-dose, marrow-ablative chemotherapeutic regimens supported by stem cell rescue have also demonstrated that a subgroup of patients with medulloblastoma who are younger than 3 years and, in some studies, younger than 5 years at the time of diagnosis can be successfully treated with chemotherapy alone.[104,106,112][Level of evidence B4]; [113]

The best survival rates using this higher-dose chemotherapy approach have been seen in patients with desmoplastic medulloblastoma and MBEN.[113]

Another treatment option for children younger than 3 years at diagnosis is high-dose chemotherapy. Results of trials using higher-dose, marrow-ablative chemotherapeutic regimens supported by stem cell rescue have also demonstrated that a subgroup of patients with medulloblastoma who are younger than 3 years and, in some studies, younger than 5 years at the time of diagnosis can be successfully treated with chemotherapy alone.[104,106,112][Level of evidence B4]; [113] › The best survival rates using this higher-dose chemotherapy approach have been seen in patients with desmoplastic medulloblastoma and MBEN.[113]

After treatment with chemotherapy without concomitant radiation therapy, patients with nondisseminated disease have achieved survival rates of nearly 90%, and patients with disseminated disease have achieved survival rates of 80%.

One study reported the outcomes of infants and young children with relapsed medulloblastoma who were initially treated without craniospinal irradiation (CSI).[114]

One study reported the outcomes of infants and young children with relapsed medulloblastoma who were initially treated without craniospinal irradiation (CSI).[114]

A substantial portion of these children were treated with CSI-based regimens and their disease was successfully salvaged.

One study reported the outcomes of infants and young children with relapsed medulloblastoma who were initially treated without craniospinal irradiation (CSI).[114]

The 3-year postrelapse survival rate was 52.4% for patients treated with curative intent.

One study reported the outcomes of infants and young children with relapsed medulloblastoma who were initially treated without craniospinal irradiation (CSI).[114]

The report found that older age at diagnosis, local relapse, and the SHH infant medulloblastoma subtype were associated with better postrelapse survival.

One study reported the outcomes of infants and young children with relapsed medulloblastoma who were initially treated without craniospinal irradiation (CSI).[114]

The addition of chemotherapy to CSI did not improve outcomes.